Abrégé

The present disclosure provides variant AAV capsid polypeptides that, when present in the capsid of an AAV vector, facilitate efficient transduction of cardiac cells, in particular human cardiac cells. Also provided are methods for preparing AAV vectors, in particular vectors that transduce cardiac cells, and in particular AAV vectors for gene therapy uses, in particular those targeting the heart or cardiac cells and tissue.

Classes IPC  ?

• C07K 14/005 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant de virus
• C07K 14/075 - Adénoviridae
• C12N 7/00 - Virus, p. ex. bactériophagesCompositions les contenantLeur préparation ou purification
• C12N 15/86 - Vecteurs viraux

Abrégé

The present disclosure relates generally to modified adeno-associated virus (AAV) capsid polypeptides and encoding nucleic acid molecules. The disclosure also relates to AAV vectors comprising the capsid polypeptides, and nucleic acid vectors (e.g. plasmids) comprising the encoding nucleic acids molecules, as well as to host cells comprising the vectors. The disclosure also relates to methods and uses of the polypeptides, encoding nucleic acids molecules, vectors and host cells.

Classes IPC  ?

• C07K 14/005 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant de virus

Abrégé

Provided herein are methods for identifying novel cap genes suitable for vectorization, utilizing selection based on homologous recombination-mediated editing of a distinct genetic basis. Also provided is the selection and production of AAV vectors that can facilitate homologous recombination-mediated gene editing in a host cell or tissue of choice. Also provided are replication-incompetent AAV and AAV libraries for use in the methods, and nucleic acid molecules to generate the replication-incompetent AAV particles and AAV libraries.

Classes IPC  ?

• C12N 15/10 - Procédés pour l'isolement, la préparation ou la purification d'ADN ou d'ARN

Abrégé

The present disclosure generally relates to methods of treating cancer and more particularly ALT- dependent cancers.

Classes IPC  ?

• A61K 31/713 - Acides nucléiques ou oligonucléotides à structure en double-hélice
• A61P 35/00 - Agents anticancéreux
• C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens

Abrégé

The present disclosure relates generally to modified adeno-associated virus (AAV) capsid polypeptides and encoding nucleic acid molecules. The disclosure also relates to AAV vectors comprising the capsid polypeptides, and nucleic acid vectors (e.g. plasmids) comprising the encoding nucleic acids molecules, as well as to host cells comprising the vectors. The disclosure also relates to methods and uses of the polypeptides, encoding nucleic acids molecules, vectors and host cells.

Classes IPC  ?

• C07K 14/005 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant de virus
• C12N 15/86 - Vecteurs viraux

Abrégé

The present invention relates to novel adeno-associated virus (AAV) variants capable of targeting cells of the peripheral nervous system. Provided herein are AAV capsid polypeptides and encoding nucleic acid molecules, the capsid polypeptides. Also provided are AAV vectors comprising the capsid polypeptides, and nucleic acid vectors (e.g. plasmids) comprising the encoding nucleic acids molecules, as well as host cells comprising the vectors. Also provided are methods and uses of the polypeptides, encoding nucleic acids molecules, vectors and host cells.

Classes IPC  ?

• C07K 14/015 - Parvoviridae, p. ex. virus de l'aleucémie féline, parvovirus humain
• A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
• A61P 25/00 - Médicaments pour le traitement des troubles du système nerveux
• C12N 15/864 - Vecteurs parvoviraux

Abrégé

The present disclosure relates generally to gene editing systems comprising an adeno-associated virus (AAV) vector comprising a cDNA donor sequence. In particular, the gene editing systems of the present disclosure integrate the cDNA donor sequence at a target locus in an intron of a gene encoding a urea cycle enzyme by non-homologous end joining (NHEJ). The disclosure also relates to methods and uses of the gene editing systems, e.g., for the treatment of urea cycle disorders.

Classes IPC  ?

• C12N 15/86 - Vecteurs viraux
• A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
• A61P 1/16 - Médicaments pour le traitement des troubles du tractus alimentaire ou de l'appareil digestif des troubles de la vésicule biliaire ou du foie, p. ex. protecteurs hépatiques, cholagogues, cholélitholytiques
• A61P 3/00 - Médicaments pour le traitement des troubles du métabolisme
• A61P 13/00 - Médicaments pour le traitement des troubles du système urinaire
• C12N 9/00 - Enzymes, p. ex. ligases (6.)ProenzymesCompositions les contenantProcédés pour préparer, activer, inhiber, séparer ou purifier des enzymes
• C12N 9/10 - Transférases (2.)
• C12N 9/22 - Ribonucléases
• C12N 15/11 - Fragments d'ADN ou d'ARNLeurs formes modifiées
• C12N 15/90 - Introduction stable d'ADN étranger dans le chromosome

Abrégé

The present disclosure relates generally to adeno-associated vims (AAV) capsid polypeptides and encoding nucleic acid molecules. The disclosure also relates to AAV vectors comprising the capsid polypeptides, and nucleic acid vectors (e.g. plasmids) comprising the encoding nucleic acids molecules, as well as to host cells comprising the vectors. The disclosure also relates to methods and uses of the polypeptides, encoding nucleic acids molecules, vectors and host cells. The disclosure includes AAV capsid polypeptides comprising a peptide modification in variable region 8 (VR VIII), where the peptide modification comprises a 7 amino acid insertion.

Classes IPC  ?

• C07K 14/005 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant de virus
• C12N 7/00 - Virus, p. ex. bactériophagesCompositions les contenantLeur préparation ou purification
• C12N 15/86 - Vecteurs viraux

Abrégé

The present disclosure relates generally to adeno-associated vims (AAV) capsid polypeptides and encoding nucleic acid molecules. The disclosure also relates to AAV vectors comprising the capsid polypeptides, and nucleic acid vectors (e.g. plasmids) comprising the encoding nucleic acids molecules, as well as to host cells comprising the vectors. The disclosure also relates to methods and uses of the polypeptides, encoding nucleic acids molecules, vectors and host cells.

Classes IPC  ?

• C07K 14/075 - Adénoviridae

Abrégé

Provided herein are AAV capsid polypeptides comprising peptide modifications relative to the wild-type AAV6 polypeptide that, when present in the capsid of an AAV vector, can facilitate homology directed repair (HDR)-mediated gene editing of human T cells. Also provided are AAV vectors comprising the capsid polypeptides, nucleic acid vectors comprising the encoding nucleic acid molecules, and host cells comprising the vectors, as well as methods of use of such AAV vectors, nucleic acid vectors and host cells.

Classes IPC  ?

• C07K 14/005 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant de virus
• A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
• A61P 37/04 - Immunostimulants
• C07K 14/015 - Parvoviridae, p. ex. virus de l'aleucémie féline, parvovirus humain
• C12N 7/00 - Virus, p. ex. bactériophagesCompositions les contenantLeur préparation ou purification
• C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
• C12N 15/86 - Vecteurs viraux
• C12N 15/90 - Introduction stable d'ADN étranger dans le chromosome

Abrégé

The present disclosure generally relates to cancer and the treatment of cancer, including the treatment of Alternative Lengthening of Telomeres (ALT) cancers. The disclosure provides methods comprising inhibiting FANCM activity or expression, such as inhibiting FANCM's interaction with RMI and/or FANCM's ATPase activity, to inhibit the growth and/or proliferation of ALT tumor cells and/or to induce death of ATL tumor cells. The methods may be practiced on patients diagnosed with an ALT tumor.

Classes IPC  ?

• A61K 38/17 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains
• C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
• A61K 31/496 - Pipérazines non condensées contenant d'autres hétérocycles, p. ex. rifampine, thiothixène ou sparfloxacine
• A61P 35/00 - Agents anticancéreux
• A61K 45/06 - Mélanges d'ingrédients actifs sans caractérisation chimique, p. ex. composés antiphlogistiques et pour le cœur
• G01N 33/50 - Analyse chimique de matériau biologique, p. ex. de sang ou d'urineTest par des méthodes faisant intervenir la formation de liaisons biospécifiques par ligandsTest immunologique

Abrégé

Provided herein are methods for identifying novel cap genes suitable for vectorization, utilizing selection based on homologous recombination-mediated editing of a distinct genetic basis. Also provided is the selection and production of AAV vectors that can facilitate homologous recombination-mediated gene editing in a host cell or tissue of choice. Also provided are replication-incompetent AAV and AAV libraries for use in the methods, and nucleic acid molecules to generate the replication-incompetent AAV particles and AAV libraries.

Classes IPC  ?

• C12N 15/10 - Procédés pour l'isolement, la préparation ou la purification d'ADN ou d'ARN
• C12N 15/86 - Vecteurs viraux

Abrégé

The present disclosure relates generally to adeno-associated virus (AAV) capsid polypeptides and encoding nucleic acid molecules. The disclosure also relates to AAV vectors comprising the capsid polypeptides, and nucleic acid vectors (e.g. plasmids) comprising the encoding nucleic acids molecules, as well as to host cells comprising the vectors. The disclosure also relates to methods and uses of the polypeptides, encoding nucleic acids molecules, vectors and host cells.

Classes IPC  ?

• C07K 14/005 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant de virus
• C12N 15/86 - Vecteurs viraux

Abrégé

e.g.e.g. plasmids) comprising the encoding nucleic acids molecules, as well as to host cells comprising the vectors. The disclosure also relates to methods and uses of the polypeptides, encoding nucleic acids molecules, vectors and host cells.

Classes IPC  ?

• C07K 14/075 - Adénoviridae
• C12N 7/00 - Virus, p. ex. bactériophagesCompositions les contenantLeur préparation ou purification
• C07K 14/005 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant de virus
• C12N 15/86 - Vecteurs viraux

Abrégé

The present disclosure relates generally to modified adeno-associated virus (AAV) capsid polypeptides and encoding nucleic acid molecules. The disclosure also relates to AAV vectors comprising the capsid polypeptides, and nucleic acid vectors (e.g. plasmids) comprising the encoding nucleic acids molecules, as well as to host cells comprising the vectors. The disclosure also relates to methods and uses of the polypeptides, encoding nucleic acids molecules, vectors and host cells.

Classes IPC  ?

• C07K 14/005 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant de virus
• C07K 14/075 - Adénoviridae
• C12N 7/00 - Virus, p. ex. bactériophagesCompositions les contenantLeur préparation ou purification
• C12N 15/86 - Vecteurs viraux

Abrégé

The present disclosure relates generally to modified adeno-associated virus (AAV) capsid polypeptides and encoding nucleic acid molecules. The disclosure also relates to AAV vectors comprising the capsid polypeptides, and nucleic acid vectors (e.g. plasmids) comprising the encoding nucleic acids molecules, as well as to host cells comprising the vectors. The disclosure also relates to methods and uses of the polypeptides, encoding nucleic acids molecules, vectors and host cells.

Classes IPC  ?

• C07K 14/075 - Adénoviridae

Abrégé

The present disclosure relates generally to methods and adeno-associated virus (AAV) vectors for in vivo transduction. The disclosure is directed to methods for producing modified AAV capsid polypeptides and AAV vectors that are suitable for transduction of cells in vivo, and methods for screening AAV vectors. The disclosure also relates to adeno-associated virus (AAV) capsid polypeptides and encoding nucleic acid molecules, AAV vectors comprising the capsid polypeptides, and nucleic acid vectors comprising the encoding nucleic acids molecules, as well as to host cells comprising the vectors. The disclosure also relates to methods and uses of the polypeptides, encoding nucleic acids molecules, vectors and host cells.

Classes IPC  ?

• C12N 15/86 - Vecteurs viraux
• C07K 14/005 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant de virus
• A61P 1/16 - Médicaments pour le traitement des troubles du tractus alimentaire ou de l'appareil digestif des troubles de la vésicule biliaire ou du foie, p. ex. protecteurs hépatiques, cholagogues, cholélitholytiques

Abrégé

The present disclosure relates to inhibitors of ZNF827 and their use alone or in combination with anti-cancer agents to treat cancer. Methods of selecting a subject for treatment with an inhibitor of ZNF827 are also detailed.

Classes IPC  ?

• A61K 31/4745 - QuinoléinesIsoquinoléines condensées en ortho ou en péri avec des systèmes hétérocycliques condensées avec des systèmes cycliques ayant l'azote comme hétéro-atome d'un cycle, p. ex. phénanthrolines
• A61P 35/00 - Agents anticancéreux
• A61K 31/047 - Composés hydroxylés, p. ex. alcoolsLeurs sels, p. ex. alcoolates ayant plusieurs groupes hydroxyle, p. ex. sorbitol
• A61K 31/519 - PyrimidinesPyrimidines hydrogénées, p. ex. triméthoprime condensées en ortho ou en péri avec des hétérocycles
• A61K 31/138 - Aryloxyalkylamines, p. ex. propranolol, tamoxifène, phénoxybenzamine
• A61K 31/4439 - Pyridines non condenséesLeurs dérivés hydrogénés contenant d'autres systèmes hétérocycliques contenant un cycle à cinq chaînons avec l'azote comme hétéro-atome du cycle, p. ex. oméprazole
• A61K 31/454 - Pipéridines non condensées, p. ex. pipérocaïne contenant d'autres systèmes hétérocycliques contenant un cycle à cinq chaînons avec l'azote comme hétéro-atome du cycle, p. ex. pimozide, dompéridone
• A61K 31/502 - PyridazinesPyridazines hydrogénées condensées en ortho ou en péri avec des systèmes carbocycliques, p. ex. cinnoline, phtalazine
• A61K 31/55 - Composés hétérocycliques ayant l'azote comme hétéro-atome d'un cycle, p. ex. guanéthidine ou rifamycines ayant des cycles à sept chaînons, p. ex. azélastine, pentylènetétrazole
• A61K 31/192 - Acides carboxyliques, p. ex. acide valproïque ayant des groupes aromatiques, p. ex. sulindac, acides 2-aryl-propioniques, acide éthacrynique
• A61K 31/506 - PyrimidinesPyrimidines hydrogénées, p. ex. triméthoprime non condensées et contenant d'autres hétérocycles
• A61K 31/155 - Amidines (), p. ex. guanidine (H2N-C(=NH)-NH2), isourée (HN=C(OH)NH2), isothiourée (HN=C(SH)-NH2)
• C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens

Abrégé

The present disclosure relates generally to adeno-associated virus (AAV) capsid polypeptides and encoding nucleic acid molecules. The disclosure also relates to AAV vectors comprising the capsid polypeptides, and nucleic acid vectors (e.g. plasmids) comprising the encoding nucleic acids molecules, as well as to host cells comprising the vectors. The disclosure also relates to methods and uses of the polypeptides, encoding nucleic acids molecules, vectors and host cells.

Classes IPC  ?

• C07K 14/005 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant de virus
• C12N 15/86 - Vecteurs viraux
• C12N 7/00 - Virus, p. ex. bactériophagesCompositions les contenantLeur préparation ou purification

Abrégé

The present disclosure relates generally to adeno-associated virus (AAV) capsid polypeptides and encoding nucleic acid molecules. The disclosure also relates to AAV vectors comprising the capsid polypeptides, and nucleic acid vectors (e.g. plasmids) comprising the encoding nucleic acids molecules, as well as to host cells comprising the vectors. The disclosure also relates to methods and uses of the polypeptides, encoding nucleic acids molecules, vectors and host cells. The disclosure includes AAV capsid polypeptides comprising a peptide modification in variable region 8 (VR VIII), where the peptide modification comprises a 7 amino acid insertion.

Classes IPC  ?

• C07K 14/005 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant de virus
• C12N 15/86 - Vecteurs viraux
• C12N 7/00 - Virus, p. ex. bactériophagesCompositions les contenantLeur préparation ou purification

Abrégé

The present disclosure relates generally to nucleic acid molecules and methods for identifying AAV vectors with desirable properties, including nucleic acid molecules and methods useful for identifying novel cap genes for vectorization, production of AAV vectors and AAV libraries.

Classes IPC  ?

• C12N 15/10 - Procédés pour l'isolement, la préparation ou la purification d'ADN ou d'ARN
• C12N 15/86 - Vecteurs viraux

Abrégé

The present invention generally relates to the use of halodiaminopyrimidine sulphonylbenzene compounds in the treatment or prevention of diseases, disorders or conditions involving a dynamin-mediated pathway.

Classes IPC  ?

• A61K 31/505 - PyrimidinesPyrimidines hydrogénées, p. ex. triméthoprime
• A61K 31/506 - PyrimidinesPyrimidines hydrogénées, p. ex. triméthoprime non condensées et contenant d'autres hétérocycles
• A61P 31/14 - Antiviraux pour le traitement des virus ARN
• A61P 11/00 - Médicaments pour le traitement des troubles du système respiratoire

Abrégé

The present disclosure relates generally to adeno-associated virus (AAV) capsid polypeptides and encoding nucleic acid molecules. The disclosure also relates to AAV vectors comprising the capsid polypeptides, and nucleic acid vectors (e.g. plasmids) comprising the encoding nucleic acids molecules, as well as to host cells comprising the vectors. The disclosure also relates to methods and uses of the polypeptides, encoding nucleic acids molecules, vectors and host cells.

Classes IPC  ?

• C07K 14/005 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant de virus
• C12N 5/071 - Cellules ou tissus de vertébrés, p. ex. cellules humaines ou tissus humains
• C12N 7/00 - Virus, p. ex. bactériophagesCompositions les contenantLeur préparation ou purification
• C12N 15/86 - Vecteurs viraux

Abrégé

The present disclosure relates generally to adeno-associated virus (AAV) capsid polypeptides and encoding nucleic acid molecules. The disclosure also relates to AAV vectors comprising the capsid polypeptides, and nucleic acid vectors (e.g. plasmids) comprising the encoding nucleic acids molecules, as well as to host cells comprising the vectors. The disclosure also relates to methods and uses of the polypeptides, encoding nucleic acids molecules, vectors and host cells.

Classes IPC  ?

• C07K 14/005 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant de virus
• C12N 15/86 - Vecteurs viraux
• C12N 5/071 - Cellules ou tissus de vertébrés, p. ex. cellules humaines ou tissus humains
• C12N 7/00 - Virus, p. ex. bactériophagesCompositions les contenantLeur préparation ou purification

Abrégé

The present disclosure relates generally to polypeptides derived from marsupial adeno-associated vims (AAV). The disclosure is also related to nucleic acid molecules encoding the polypeptides, and vectors comprising the nucleic acid molecules, and AAV vectors comprising the polypeptides. The disclosure also relates to uses of the nucleic acid molecules, polypeptides and AAV vectors, such as for capsid diversification.

Classes IPC  ?

• C07K 14/075 - Adénoviridae
• C12N 15/10 - Procédés pour l'isolement, la préparation ou la purification d'ADN ou d'ARN
• C12N 15/86 - Vecteurs viraux

Abrégé

in vivoin vivoin vivo, and methods for screening AAV vectors. The disclosure also relates to adeno-associated virus (AAV) capsid polypeptides and encoding nucleic acid molecules, AAV vectors comprising the capsid polypeptides, and nucleic acid vectors comprising the encoding nucleic acids molecules, as well as to host cells comprising the vectors. The disclosure also relates to methods and uses of the polypeptides, encoding nucleic acids molecules, vectors and host cells.

Classes IPC  ?

• A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
• C07K 14/015 - Parvoviridae, p. ex. virus de l'aleucémie féline, parvovirus humain
• A61P 1/16 - Médicaments pour le traitement des troubles du tractus alimentaire ou de l'appareil digestif des troubles de la vésicule biliaire ou du foie, p. ex. protecteurs hépatiques, cholagogues, cholélitholytiques

Abrégé

The present disclosure relates to inhibitors of ZNF827 and their use alone or in combination with anti-cancer agents to treat cancer. Methods of selecting a subject for treatment with an inhibitor of ZNF827 are also detailed.

Classes IPC  ?

• A61K 31/7105 - Acides ribonucléiques naturels, c.-à-d. contenant uniquement des riboses liés à l'adénine, la guanine, la cytosine ou l'uracile et ayant des liaisons 3'-5' phosphodiester
• A61P 35/00 - Agents anticancéreux
• A61K 39/395 - AnticorpsImmunoglobulinesImmunsérum, p. ex. sérum antilymphocitaire
• A61K 38/02 - Peptides à nombre indéterminé d'amino-acidesLeurs dérivés

Abrégé

The present disclosure generally relates to cancer and the treatment of cancer, including the treatment of Alternative Lengthening of Telomeres (ALT) cancers. The disclosure provides methods comprising inhibiting FANCM activity or expression, such as inhibiting FANCM's interaction with RMI and/or FANCM's ATPase activity, to inhibit the growth and/or proliferation of ALT tumor cells and/or to induce death of ATL tumor cells. The methods may be practiced on patients diagnosed with an ALT tumor.

Classes IPC  ?

• C12N 15/00 - Techniques de mutation ou génie génétiqueADN ou ARN concernant le génie génétique, vecteurs, p. ex. plasmides, ou leur isolement, leur préparation ou leur purificationUtilisation d'hôtes pour ceux-ci

Abrégé

The present disclosure generally relates to cancer and the treatment of cancer, including the treatment of Alternative Lengthening of Telomeres (ALT) cancers. The disclosure provides methods comprising inhibiting FANCM activity or expression, such as inhibiting FANCM's interaction with RMI and/or FANCM's ATPase activity, to inhibit the growth and/or proliferation of ALT tumor cells and/or to induce death of ATL tumor cells. The methods may be practiced on patients diagnosed with an ALT tumor.

Classes IPC  ?

• A61K 39/395 - AnticorpsImmunoglobulinesImmunsérum, p. ex. sérum antilymphocitaire
• C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
• A61K 31/4439 - Pyridines non condenséesLeurs dérivés hydrogénés contenant d'autres systèmes hétérocycliques contenant un cycle à cinq chaînons avec l'azote comme hétéro-atome du cycle, p. ex. oméprazole
• A61K 31/7088 - Composés ayant au moins trois nucléosides ou nucléotides
• A61K 31/713 - Acides nucléiques ou oligonucléotides à structure en double-hélice
• A61K 38/17 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains
• A61K 38/46 - Hydrolases (3)
• A61P 35/00 - Agents anticancéreux
• C07K 7/08 - Peptides linéaires ne contenant que des liaisons peptidiques normales ayant de 12 à 20 amino-acides
• C07K 14/47 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains provenant de vertébrés provenant de mammifères
• C12N 15/00 - Techniques de mutation ou génie génétiqueADN ou ARN concernant le génie génétique, vecteurs, p. ex. plasmides, ou leur isolement, leur préparation ou leur purificationUtilisation d'hôtes pour ceux-ci
• C12N 15/63 - Introduction de matériel génétique étranger utilisant des vecteursVecteurs Utilisation d'hôtes pour ceux-ciRégulation de l'expression
• C12Q 1/00 - Procédés de mesure ou de test faisant intervenir des enzymes, des acides nucléiques ou des micro-organismesCompositions à cet effetProcédés pour préparer ces compositions
• C12Q 1/68 - Procédés de mesure ou de test faisant intervenir des enzymes, des acides nucléiques ou des micro-organismesCompositions à cet effetProcédés pour préparer ces compositions faisant intervenir des acides nucléiques

Abrégé

The present disclosure relates generally to nucleic acid molecules and methods for identifying AAV vectors with desirable properties, including nucleic acid molecules and methods useful for identifying novel cap genes for vectorization, production of AAV vectors and AAV libraries.

Classes IPC  ?

• C12N 7/00 - Virus, p. ex. bactériophagesCompositions les contenantLeur préparation ou purification
• C40B 40/02 - Bibliothèques contenues ou présentées dans des micro-organismes, p. ex. des bactéries ou des cellules animalesBibliothèques contenues ou présentées dans des vecteurs, p. ex. des plasmidesBibliothèques contenant uniquement des micro-organismes ou des vecteurs

Abrégé

Provided herein are methods for facilitating or inducing stable transgene integration and expression in a proliferating cell, comprising administering to the cell (i) a recombinant AAV (rAAV) vector comprising the transgene flanked by transposon-derived inverted terminal repeat sequences, which sequences are in turn flanked by AAV-derived inverted terminal repeat regions, and (ii) a source of a transposase that recognises said transposon-derived inverted terminal repeat sequences and directs the genomic integration of the transgene into the genome of the proliferating cell. Also provide are methods and transgene delivery systems for the treatment or prevention of diseases affecting, associated with or characterised by proliferating cells.

Classes IPC  ?

• A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
• C12N 15/86 - Vecteurs viraux
• C12N 9/00 - Enzymes, p. ex. ligases (6.)ProenzymesCompositions les contenantProcédés pour préparer, activer, inhiber, séparer ou purifier des enzymes
• C12N 9/10 - Transférases (2.)
• C12N 9/12 - Transférases (2.) transférant des groupes contenant du phosphore, p. ex. kinases (2.7)
• C12N 9/14 - Hydrolases (3.)

Abrégé

Polynucleotides and vectors can be used for the expression of a transgene in cells, such as liver cells. The expression of the transgene from the polynucleotides and vectors can be useful in gene therapy. Various methods can be used for expressing the transgene from the polynucleotides and vectors in liver cells.

Classes IPC  ?

• C12N 15/67 - Méthodes générales pour favoriser l'expression
• A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
• C12N 9/10 - Transférases (2.)
• C12N 15/86 - Vecteurs viraux

Abrégé

The present disclosure relates generally to polypeptides derived from marsupial adeno-associated virus (AAV). The disclosure is also related to nucleic acid molecules encoding the polypeptides, and vectors comprising the nucleic acid molecules, and AAV vectors comprising the polypeptides. The disclosure also relates to uses of the nucleic acid molecules, polypeptides and AAV vectors, such as for capsid diversification.

Classes IPC  ?

• C07K 14/015 - Parvoviridae, p. ex. virus de l'aleucémie féline, parvovirus humain
• C12N 7/00 - Virus, p. ex. bactériophagesCompositions les contenantLeur préparation ou purification
• C12N 15/86 - Vecteurs viraux
• C07K 19/00 - Peptides hybrides
• A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique

Abrégé

The present disclosure relates generally to methods for modifying the sequence of a polynucleotide, or the sequence of a plurality of polynucleotides, so as to produce a modified polynucleotide(s) with altered codon usage. The disclosure also relates to methods for expressing the modified polynucleotides and methods for producing chimeric genes from the modified polynucleotides.

Classes IPC  ?

• C12N 15/10 - Procédés pour l'isolement, la préparation ou la purification d'ADN ou d'ARN
• C40B 40/08 - Bibliothèques comprenant de l'ARN ou de l'ADN codant des protéines, p. ex. bibliothèques de gènes
• G16B 35/10 - Conception de bibliothèques

Abrégé

Polypeptides are encoded by an adeno-associated virus (AAV)-derived endogenous viral element (mAAV-EVE1) found within the germline of numerous closely-related marsupial species. Nucleic acid molecules encode the polypeptides. Vectors can include the nucleic acid molecules, and recombinant AAV virions can include the polypeptides. A chimeric capsid protein can also include an MAAV-EVE1 polypeptide.

Classes IPC  ?

• C07K 14/005 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant de virus
• C07K 14/015 - Parvoviridae, p. ex. virus de l'aleucémie féline, parvovirus humain
• C12N 15/86 - Vecteurs viraux
• C12N 15/10 - Procédés pour l'isolement, la préparation ou la purification d'ADN ou d'ARN
• A61K 38/00 - Préparations médicinales contenant des peptides
• A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique

Abrégé

This invention relates generally to methods for assessing telomeres, including methods of measuring the length of a telomere and methods for detecting extension of a telomere and methods for measuring telomere extension, and associated systems.

Classes IPC  ?

• G01N 33/00 - Recherche ou analyse des matériaux par des méthodes spécifiques non couvertes par les groupes
• C12Q 1/68 - Procédés de mesure ou de test faisant intervenir des enzymes, des acides nucléiques ou des micro-organismesCompositions à cet effetProcédés pour préparer ces compositions faisant intervenir des acides nucléiques
• G01N 33/53 - Tests immunologiquesTests faisant intervenir la formation de liaisons biospécifiquesMatériaux à cet effet

Abrégé

The present invention is directed to polynucleotides and vectors for the expression of a transgene in liver cells. The present invention is also directed to various uses of the polynucleotides and vectors, such as in gene therapy, and methods for expressing the transgene from the polynucleotides and vectors in liver cells. In particular the invention relates to polynucleotides and vectors comprising a human ornithine transcarbamylase (hOTC) enhancer and a liver specific promoter. In particular embodiments the transgene is ornithine transcarbamylase and the vectors are used in the treatment or prevention of ornithine transcarbamylase deficiency or hyperammonemia.

Classes IPC  ?

• C12N 15/63 - Introduction de matériel génétique étranger utilisant des vecteursVecteurs Utilisation d'hôtes pour ceux-ciRégulation de l'expression
• C12N 15/67 - Méthodes générales pour favoriser l'expression
• C12N 15/85 - Vecteurs ou systèmes d'expression spécialement adaptés aux hôtes eucaryotes pour cellules animales

Abrégé

The present disclosure relates generally to promoters derived from the AAV anti-sense strand, their use in the expression of one or more heterologous coding sequences, and isolated polynucleotides, vectors and recombinant viruses comprising the promoters. The present disclosure also relates to enhancers derived from the AAV anti-sense strand, their use in increasing the expression of one or more heterologous coding sequences, and isolated polynucleotides, vectors and recombinant viruses comprising the enhancers.

Classes IPC  ?

• C12N 15/864 - Vecteurs parvoviraux
• C12N 15/85 - Vecteurs ou systèmes d'expression spécialement adaptés aux hôtes eucaryotes pour cellules animales
• C12N 15/11 - Fragments d'ADN ou d'ARNLeurs formes modifiées
• C12N 15/63 - Introduction de matériel génétique étranger utilisant des vecteursVecteurs Utilisation d'hôtes pour ceux-ciRégulation de l'expression
• A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
• C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
• C12N 15/86 - Vecteurs viraux
• C12N 15/867 - Vecteurs rétroviraux
• C12N 15/869 - Vecteurs herpèsviraux

Produits et services

Software and computer programs including, but not limited to, software and computer programs in respect of medical, proteomic and genetic analysis and testing; computer hardware and computer apparatus including computer hardware and computer apparatus for use in respect of medical, proteomic and genetic analysis and testing; mass-spectrometers in this class; databases; databases (electronic publications). Printed matter; printed publications including journals, leaflets, pamphlets, booklets and papers in respect to medical research and development services, genetics and proteomics. Charitable collections; financial services including fundraising and charitable fundraising; financial sponsorship services; funding of scientific and medical research; fundraising activities namely fundraising services relating to the conduct of auctions and other sales, organisation of fundraising events, charity services, sponsorships and accreditation, special event days; advisory, consultancy and information services in respect of all of the aforementioned services; charitable fundraising of all of the aforementioned services. Education services including education services in respect of medical, proteomic and genetic analysis and testing and medical research and development; training services including training services in respect of medical, proteomic and genetic analysis and testing and medical research and development; publishing by electronic means; publishing of books, documents, newsletters and printed matter including those in respect of medical, proteomic and genetic analysis and testing and medical research and development; advisory, consultancy and information services in respect of all of the aforementioned services. Research including, but not limited to, medical research; medical research (scientific research for medical purposes); scientific research and analysis, including for medical purposes; analysis of tissues for medical research; research laboratories; biological research; biotechnology research; genetic research; development of testing methods and testing methodologies including in respect to medical research; development of test equipment for medical research; technical research; database design; scientific research relating to genetics; advisory, consultancy and information services in respect of all of the aforementioned services. Medical services including medical health assessment services; health care services; health screening services; health risk assessment surveys; arranging of medical treatment; services for the preparation of medical reports; conducting of medical examinations; genetic testing for medical purposes; advisory, consultancy and information services in respect of all of the aforementioned services.

Produits et services

Software and computer programs including, but not limited to, software and computer programs in respect of medical, proteomic and genetic analysis and testing; computer hardware and computer apparatus including computer hardware and computer apparatus for use in respect of medical, proteomic and genetic analysis and testing; mass-spectrometers in this class; databases; databases (electronic publications). Printed matter; printed publications including journals, leaflets, pamphlets, booklets and papers in respect to medical research and development services, genetics and proteomics. Charitable collections; financial services including fundraising and charitable fundraising; financial sponsorship services; funding of scientific and medical research; fundraising activities namely fundraising services relating to the conduct of auctions and other sales, organisation of fundraising events, charity services, sponsorships and accreditation, special event days; advisory, consultancy and information services in respect of all of the aforementioned services; charitable fundraising of all the aforementioned services. Education services including education services in respect of medical, proteomic and genetic analysis and testing and medical research and development; training services including training services in respect of medical, proteomic and genetic analysis and testing and medical research and development; publishing by electronic means; publishing of books, documents, newsletters and printed matter including those in respect of medical, proteomic and genetic analysis and testing and medical research and development; advisory, consultancy and information services in respect of all of the aforementioned services. Research including, but not limited to, medical research; medical research (scientific research for medical purposes); scientific research and analysis, including for medical purposes; analysis of tissues for medical research; research laboratories; biological research; biotechnology research; genetic research; development of testing methods and testing methodologies including in respect to medical research; development of test equipment for medical research; technical research; database design; scientific research relating to genetics; advisory, consultancy and information services in respect of all of the aforementioned services. Medical services including medical health assessment services; health care services; health screening services; health risk assessment surveys; arranging of medical treatment; conducting of medical examinations; genetic testing for medical purposes; advisory, consultancy and information services in respect of all of the aforementioned services.

Abrégé

The present disclosure relates generally to polypeptides encoded by an adeno-associated virus (AAV)-derived endogenous viral element (mAAV-EVEl) found within the germline of numerous closely-related marsupial species. The disclosure is also related to nucleic acid molecules encoding the polypeptides, vectors comprising the nucleic acid molecules and recombinant AAV virions comprising polypeptides. The disclosure also relates to uses of nucleic acid molecules, vectors and recombinant AAV virions.

Classes IPC  ?

• C07K 14/015 - Parvoviridae, p. ex. virus de l'aleucémie féline, parvovirus humain
• A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
• C12N 15/86 - Vecteurs viraux

Produits et services

Software and computer programs including, but not limited to, software and computer programs in respect of medical, proteomic and genetic analysis and testing; computer hardware and computer apparatus including computer hardware and computer apparatus for use in respect of medical, proteomic and genetic analysis and testing; mass-spectrometers in this class; databases; databases (electronic publications). Printed matter; printed publications including journals, leaflets, pamphlets, booklets and papers in respect to medical research and development services, genetics and proteomics. Charitable collections; financial services including fundraising and charitable fundraising; financial sponsorship services; funding of scientific and medical research; fundraising activities namely fundraising services relating to the conduct of auctions and other sales, organisation of fundraising events, charity services, sponsorships and accreditation, special event days; advisory, consultancy and information services in respect of all of the aforementioned services; charitable fundraising of all of the aforementioned services. Education services including education services in respect of medical, proteomic and genetic analysis and testing and medical research and development; training services including training services in respect of medical, proteomic and genetic analysis and testing and medical research and development; publishing by electronic means; publishing of books, documents, newsletters and printed matter including those in respect of medical, proteomic and genetic analysis and testing and medical research and development; advisory, consultancy and information services in respect of all of the aforementioned services. Research including, but not limited to, medical research; medical research (scientific research for medical purposes); scientific research and analysis, including for medical purposes; analysis of tissues for medical research; research laboratories; biological research; biotechnology research; genetic research; development of testing methods and testing methodologies including in respect to medical research; development of test equipment for medical research; technical research; database design; scientific research relating to genetics; advisory, consultancy and information services in respect of all of the aforementioned services. Medical services including medical health assessment services; health care services; health screening services; health risk assessment surveys; arranging of medical treatment; conducting of medical examinations; genetic testing for medical purposes; advisory, consultancy and information services in respect of all of the aforementioned services.

Produits et services

(1) Software and computer programs in respect of medical, proteomic and genetic analysis and testing; computer hardware for use in respect of medical, proteomic and genetic analysis and testing; mass-spectrometers; downloadable databases in the fields of medicine, proteonomics, molecules and genetics; electronic publications, both downloadable and recorded on computer media, namely magazines, journals, newspapers, manuals, and e-books in the fields of medicine, proteomics, molecules and genetics (2) Printed publications, namely journals, leaflets, pamphlets, booklets and papers in respect to medical research and development services, genetics and proteomics (1) Charitable collections; financial services namely fundraising and charitable fundraising; fundraising activities namely fundraising services relating to the conduct of auctions and other sales, organisation of fundraising events, charity services, sponsorships and accreditation, special event days; advisory, consultancy and information services in the fields of fundraising and financial sponsorship for medical research, proteomic research, molecular research and genetic research (2) Education services namely education services in respect of medical, proteomic and genetic analysis and testing and medical research and development; training services namely training services in respect of medical, proteomic and genetic analysis and testing and medical research and development; publishing of books, documents, newsletters and printed matter in the fields of medical, proteomic and genetic analysis and testing and medical research and development and advisory, consultancy and information services relating thereto (3) Research, namely medical research and advisory, consultancy and information services in respect of all of the aforementioned services; medical research (scientific research for medical purposes) and advisory, consultancy and information services in respect of all of the aforementioned services; scientific research and analysis, namely for medical purposes and advisory, consultancy and information services in respect of all of the aforementioned services; analysis of tissues for medical research and advisory, consultancy and information services in respect of all of the aforementioned services; biotechnology research and advisory, consultancy and information services in respect of all of the aforementioned services; genetic research and advisory, consultancy and information services in respect of all of the aforementioned services; development of testing methods and testing methodologies in respect to medical research and advisory, consultancy and information services in respect of all of the aforementioned services; development of test equipment for medical research and advisory, consultancy and information services in respect of all of the aforementioned services; technical research in the fields of medicine, proteomics, biology, biotechnology and genetics and advisory, consultancy and information services in respect of all of the aforementioned services; database design and advisory, consultancy and information services in respect of all of the aforementioned services; scientific research relating to genetics and advisory, consultancy and information services in respect of all of the aforementioned services (4) Medical services namely medical health assessment services and advisory, consultancy and information services in respect thereto; health care services, namely medical treatment of cancer, health care diagnosis of diseases, and advisory, consultancy and information services in respect thereto; health screening services in the field of oncology testing, proteomic testing, molecular testing and genetic testing, and advisory, consultancy and information services in respect thereto; health risk assessment surveys and advisory, consultancy and information services in respect thereto; arranging of medical treatment and advisory, consultancy and information services in respect thereto; compilation of medical reports and advisory, consultancy and information services in respect thereto; conducting of medical examinations and advisory, consultancy and information services in respect thereto; genetic testing for medical purposes and advisory, consultancy and information services in respect thereto

Produits et services

(1) Software and computer programs in respect of medical, proteomic and genetic analysis and testing; computer hardware for use in respect of medical, proteomic and genetic analysis and testing; mass-spectrometers; downloadable databases in the fields of medicine, proteonomics, molecules and genetics; electronic publications, both downloadable and recorded on computer media, namely magazines, journals, newspapers, manuals, and e-books in the fields of medicine, proteomics, molecules and genetics (2) Printed publications, namely journals, leaflets, pamphlets, booklets and papers in respect to medical research and development services, genetics and proteomics (1) Charitable collections; financial services namely fundraising and charitable fundraising; fundraising activities namely fundraising services relating to the conduct of auctions and other sales, organisation of fundraising events, charity services, sponsorships and accreditation, special event days; advisory, consultancy and information services in the fields of fundraising and financial sponsorship for medical research, proteomic research, molecular research and genetic research (2) Education services namely education services in respect of medical, proteomic and genetic analysis and testing and medical research and development; training services namely training services in respect of medical, proteomic and genetic analysis and testing and medical research and development; publishing of books, documents, newsletters and printed matter in the fields of medical, proteomic and genetic analysis and testing and medical research and development and advisory, consultancy and information services relating thereto (3) Research, namely medical research and advisory, consultancy and information services in respect of all of the aforementioned services; medical research (scientific research for medical purposes) and advisory, consultancy and information services in respect of all of the aforementioned services; scientific research and analysis, namely for medical purposes and advisory, consultancy and information services in respect of all of the aforementioned services; analysis of tissues for medical research and advisory, consultancy and information services in respect of all of the aforementioned services; biotechnology research and advisory, consultancy and information services in respect of all of the aforementioned services; genetic research and advisory, consultancy and information services in respect of all of the aforementioned services; development of testing methods and testing methodologies in respect to medical research and advisory, consultancy and information services in respect of all of the aforementioned services; development of test equipment for medical research and advisory, consultancy and information services in respect of all of the aforementioned services; technical research in the fields of medicine, proteomics, biology, biotechnology and genetics and advisory, consultancy and information services in respect of all of the aforementioned services; database design and advisory, consultancy and information services in respect of all of the aforementioned services; scientific research relating to genetics and advisory, consultancy and information services in respect of all of the aforementioned services (4) Medical services namely medical health assessment services and advisory, consultancy and information services in respect thereto; health care services, namely medical treatment of cancer, health care diagnosis of diseases, and advisory, consultancy and information services in respect thereto; health screening services in the field of oncology testing, proteomic testing, molecular testing and genetic testing, and advisory, consultancy and information services in respect thereto; health risk assessment surveys and advisory, consultancy and information services in respect thereto; arranging of medical treatment and advisory, consultancy and information services in respect thereto; compilation of medical reports and advisory, consultancy and information services in respect thereto; conducting of medical examinations and advisory, consultancy and information services in respect thereto; genetic testing for medical purposes and advisory, consultancy and information services in respect thereto

Produits et services

(1) Software and computer programs in respect of medical, proteomic and genetic analysis and testing; computer hardware for use in respect of medical, proteomic and genetic analysis and testing; mass-spectrometers; downloadable databases in the fields of medicine, proteonomics, molecules and genetics; electronic publications, both downloadable and recorded on computer media, namely magazines, journals, newspapers, manuals, and e-books in the fields of medicine, proteomics, molecules and genetics (2) Printed publications, namely journals, leaflets, pamphlets, booklets and papers in respect to medical research and development services, genetics and proteomics (1) Charitable collections; financial services namely fundraising and charitable fundraising; fundraising activities namely fundraising services relating to the conduct of auctions and other sales, organisation of fundraising events, charity services, sponsorships and accreditation, special event days; advisory, consultancy and information services in the fields of fundraising and financial sponsorship for medical research, proteomic research, molecular research and genetic research (2) Education services namely education services in respect of medical, proteomic and genetic analysis and testing and medical research and development; training services namely training services in respect of medical, proteomic and genetic analysis and testing and medical research and development; publishing of books, documents, newsletters and printed matter in the fields of medical, proteomic and genetic analysis and testing and medical research and development and advisory, consultancy and information services relating thereto (3) Research, namely medical research and advisory, consultancy and information services in respect of all of the aforementioned services; medical research (scientific research for medical purposes) and advisory, consultancy and information services in respect of all of the aforementioned services; scientific research and analysis, namely for medical purposes and advisory, consultancy and information services in respect of all of the aforementioned services; analysis of tissues for medical research and advisory, consultancy and information services in respect of all of the aforementioned services; biotechnology research and advisory, consultancy and information services in respect of all of the aforementioned services; genetic research and advisory, consultancy and information services in respect of all of the aforementioned services; development of testing methods and testing methodologies in respect to medical research and advisory, consultancy and information services in respect of all of the aforementioned services; development of test equipment for medical research and advisory, consultancy and information services in respect of all of the aforementioned services; technical research in the fields of medicine, proteomics, biology, biotechnology and genetics and advisory, consultancy and information services in respect of all of the aforementioned services; database design and advisory, consultancy and information services in respect of all of the aforementioned services; scientific research relating to genetics and advisory, consultancy and information services in respect of all of the aforementioned services (4) Medical services namely medical health assessment services and advisory, consultancy and information services in respect thereto; health care services, namely medical treatment of cancer, health care diagnosis of diseases, and advisory, consultancy and information services in respect thereto; health screening services in the field of oncology testing, proteomic testing, molecular testing and genetic testing, and advisory, consultancy and information services in respect thereto; health risk assessment surveys and advisory, consultancy and information services in respect thereto; arranging of medical treatment and advisory, consultancy and information services in respect thereto; compilation of medical reports and advisory, consultancy and information services in respect thereto; conducting of medical examinations and advisory, consultancy and information services in respect thereto; genetic testing for medical purposes and advisory, consultancy and information services in respect thereto

Produits et services

Software and computer programs for use in medical, proteomic, molecular and genetic analysis and testing; Computer hardware for use in medical, proteomic, molecular and genetic analysis and testing; Mass-spectrometers; Downloadable databases in the fields of medicine, proteomics, molecules and genetics; Electronic publications, both downloadable and recorded on computer media, namely, magazines, journals, newspapers, manuals, and e-books in the fields of medicine, proteomics, molecules and genetics Printed matter, namely, printed publications, namely, journals, leaflets, pamphlets, booklets, and research reports in the fields of medical research and development services, genetics, molecules and proteomics Charitable fundraising; Financial services, namely, funding research in the fields of medical research, genetic research, molecular research and proteomic research; Fundraising and charitable fundraising; Financial sponsorship of medical research, genetic research, molecular research and proteomic research; Funding of scientific and medical research; Fundraising activities to support medical research, proteomic research, molecular research and genetic research; Advisory, consultancy, and information services in the fields of fundraising and financial sponsorship for medical research, proteomic research, molecular research and genetic research Education services, namely, mentoring, tutoring, classes, seminars, and workshops in the fields of medical, proteomic, molecular and genetic analysis and testing and medical research and development; Training services in the fields of medical, proteomic, molecular and genetic analysis and testing and medical research and development; Publishing of electronic publications; Publishing of books, documents, newsletters, and printed matter including those in respect of medical, proteomic, molecular and genetic analysis and testing and medical research and development; Advisory, consultancy, and information services in the fields of education and publishing Research, namely, medical research, proteomic research, molecular research and genetic research; Scientific research and analysis, including for medical purposes; Analysis of tissues for medical research; Research laboratories; Biological research; Biotechnology research; Genetic research; Development of testing methods and testing methodologies in the fields of medical research, proteomic research, molecular research and genetic research; Development of test equipment for medical research; Technical research in the fields of medicine, proteomics, biology, biotechnology, and genetics; Database design; Scientific research relating to genetics; Advisory, consultancy, and information services in the fields of medical research, scientific research, proteomic research, molecular research and genetic research Medical services including medical health assessment services; Medical services, namely, genetic, proteomic and molecular screening and testing for medical purposes; Medical services, namely, testing physical tumors; health care services; Health screening services; Health risk assessment surveys; Arranging of medical treatment; Conducting of medical examinations; Genetic testing for medical purposes; Advisory, consultancy and information services in the fields of medicine and healthcare

Produits et services

Software and computer programs for use in medical, proteomic, molecular and genetic analysis and testing; Computer hardware for use in medical, proteomic, molecular and genetic analysis and testing; Scientific apparatus, namely, mass-spectrometers; Downloadable databases in the fields of medicine, proteomics, molecules and genetics; Electronic publications, both downloadable and recorded on computer media, namely, magazines, journals, newspapers, manuals, and e-books in the fields of medicine, proteomics, molecules and genetics Printed matter, namely, printed publications, namely, journals, leaflets, pamphlets, booklets, and research reports in the fields of medical research and development services, genetics, molecules and proteomics Charitable fundraising; Financial services, namely, funding research in the fields of medical research, genetic research, molecular research and proteomic research; Fundraising services, by means of raising funds for scientific research, proteomic research, molecular research, genetic research and medical research; Financial sponsorship of medical research, genetic research, molecular research and proteomic research; Funding of scientific and medical research; Fundraising services by means of organizing special events to support medical research, proteomic research, molecular research and genetic research; Advisory, consultancy, and information services in the fields of fundraising and financial sponsorship for medical research, proteomic research, molecular research and genetic research Education services, namely, mentoring, tutoring, classes, seminars, and workshops in the fields of medical, proteomic, molecular and genetic analysis and testing and medical research and development; Training services in the fields of medical, proteomic, molecular and genetic analysis and testing and medical research and development; Publishing of electronic publications; Publishing of books, documents, newsletters, and printed matter in the fields of medical diagnosis, medical treatment, medical, proteomic, molecular and genetic analysis and testing, and medical research and development; Advisory, consultancy, and information services in the fields of education and publishing Research, namely, medical research, proteomic research, molecular research and genetic research; Scientific and technological services, namely, scientific research and analysis in the field of medicine medical diagnosis, medical treatment, proteomics, biology, biotechnology and genetics; Analysis of tissues for medical research; Research laboratories; Biological research; Biotechnology research; Genetic research; Development of testing methods and testing methodologies in the fields of medical research, proteomic research, molecular research and genetic research; Development of test equipment for medical research; Technical research in the fields of medicine, proteomics, biology, biotechnology, and genetics; Database design; Scientific research relating to genetics; Advisory, consultancy, and information services in the fields of medical research, scientific research, proteomic research, molecular research and genetic research Medical services, namely, medical health assessment services; Medical services, namely, genetic, proteomic and molecular screening and testing for medical purposes; Medical services, namely, testing physical tumors; Health care services, namely, medical treatment of cancer, health care diagnosis of diseases services, health assessment services, testing of physical tumors, and genetic, proteomic, and molecular screening and testing; Health screening services in the field of oncology testing, proteomic testing, molecular testing and genetic testing; Health risk assessment surveys; Arranging of medical treatment; Conducting of medical examinations; Genetic testing for medical purposes; Advisory, consultancy and information services in the fields of medicine and healthcare

Abrégé

The present disclosure relates generally to promoters derived from the AAV anti-sense strand, their use in the expression of one or more heterologous coding sequences, and isolated polynucleotides, vectors and recombinant viruses comprising the promoters. The present disclosure also relates to enhancers derived from the AAV anti-sense strand, their use in increasing the expression of one or more heterologous coding sequences, and isolated polynucleotides, vectors and recombinant viruses comprising the enhancers.

Classes IPC  ?

• C12N 15/63 - Introduction de matériel génétique étranger utilisant des vecteursVecteurs Utilisation d'hôtes pour ceux-ciRégulation de l'expression
• C12N 15/67 - Méthodes générales pour favoriser l'expression

Abrégé

Provided herein are methods for facilitating or inducing stable transgene integration and expression in a proliferating cell, comprising administering to the cell (i) a recombinant AAV (rAAV) vector comprising the transgene flanked by transposon-derived inverted terminal repeat sequences, which sequences are in turn flanked by AAV-derived inverted terminal repeat regions, and (ii) a source of a transposase that recognises said transposon-derived inverted terminal repeat sequences and directs the genomic integration of the transgene into the genome of the proliferating cell. Also provided are methods and transgene delivery systems for the treatment or prevention of diseases affecting, associated with or characterised by proliferating cells, including paediatric liver diseases, bone marrow diseases and cancer.

Classes IPC  ?

• C12N 15/86 - Vecteurs viraux
• A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
• C12N 15/90 - Introduction stable d'ADN étranger dans le chromosome

Abrégé

Provided herein are methods for facilitating or inducing stable transgene integration and expression in a proliferating cell, comprising administering to the cell (i) a recombinant AAV (rAAV) vector comprising the transgene flanked by transposon-derived inverted terminal repeat sequences, which sequences are in turn flanked by AAV-derived inverted terminal repeat regions, and (ii) a source of a transposase that recognises said transposon-derived inverted terminal repeat sequences and directs the genomic integration of the transgene into the genome of the proliferating cell. Also provided are methods and transgene delivery systems for the treatment or prevention of diseases affecting, associated with or characterised by proliferating cells, including paediatric liver diseases, bone marrow diseases and cancer.

Classes IPC  ?

• C12N 15/86 - Vecteurs viraux
• C12N 15/90 - Introduction stable d'ADN étranger dans le chromosome
• A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique

Abrégé

Inhibitors are provided for inhibiting the activity of clathrin. In particular, binding site(s) on the clathrin terminal doman (TD) for binding inhibitors have been identified. The binding sites are defined by amino acid(s) in the group Ile 52, Ile 62, Ile 80, Phe 91, and Ile 93 of the clathrin TD (SEQ ID No. 1). In at least some forms, the binding site may be further defined by amino acid(s) in the group Ile 66, Arg 64, Leu 82 andf Lys 96 of SEQ ID No. 1, or by Val 50 of SEQ ID No.1. There are also provided methods for prophylaxis or treatment of disease and conditions responsive to the inhibition of clathrin.

Classes IPC  ?

• A61K 31/00 - Préparations médicinales contenant des ingrédients actifs organiques
• A61P 31/00 - Agents anti-infectieux, c.-à-d. antibiotiques, antiseptiques, chimiothérapeutiques
• A61P 25/00 - Médicaments pour le traitement des troubles du système nerveux
• C40B 30/02 - Criblage par ordinateur (in silico)
• C40B 50/02 - Conception de bibliothèques par ordinateur (in silico) ou conception mathématique de bibliothèques
• G06F 19/18 - pour la génomique ou la protéomique fonctionnelle, p.ex. associations génotype-phénotype, déséquilibre de liaison, mutagénèse, génotypage ou annotation génomique, interactions protéines-protéines ou interactions protéines-acides nucléiques
• G01N 33/15 - Préparations médicinales
• C07C 13/547 - Hydrocarbures polycycliques ou leurs dérivés hydrocarbonés acycliques à cycles condensés à trois cycles condensés un cycle au moins n'étant pas un cycle à six chaînons, les autres cycles étant au plus des cycles à six chaînons
• C07C 13/573 - Hydrocarbures polycycliques ou leurs dérivés hydrocarbonés acycliques à cycles condensés à trois cycles condensés à trois cycles à six chaînons
• C07C 13/66 - Hydrocarbures polycycliques ou leurs dérivés hydrocarbonés acycliques à cycles condensés à plus de trois cycles condensés le système cyclique condensé ne contenant que quatre cycles
• C07D 221/14 - Aza-phénalènes, p. ex. naphtalimide-1, 8
• C07D 221/16 - Systèmes cycliques à trois cycles contenant des carbocycles autres que des cycles à six chaînons
• C07D 277/20 - Composés hétérocycliques contenant des cycles thiazole-1, 3 ou thiazole-1, 3 hydrogénés non condensés avec d'autres cycles comportant deux ou trois liaisons doubles entre chaînons cycliques ou entre chaînons cycliques et chaînons non cycliques
• C07D 277/36 - Atomes de soufre
• C07D 277/38 - Atomes d'azote
• C07D 277/34 - Atomes d'oxygène

Abrégé

The invention relates to methods and assays for the detection of active Alternative Lengthening of Telomeres (ALT) activity in cells. The methods and assays involve detecting or assaying for partially double-stranded telomeric circles wherein the presence of said circles is specific for cells comprising an active ALT mechanism. In some embodiments the methods find application in, inter alia, determining the level of ALT activity in a cell, determining the ALT status of a cancer in a subject, diagnosing and/or treating disease, determining disease status, analysis of treatment efficacy, and the identification of novel therapeutic agents.

Classes IPC  ?

• C12Q 1/68 - Procédés de mesure ou de test faisant intervenir des enzymes, des acides nucléiques ou des micro-organismesCompositions à cet effetProcédés pour préparer ces compositions faisant intervenir des acides nucléiques

Produits et services

Chemicals for use in the pharmaceutical industry; chemical reagents for research; laboratory chemicals for scientific use; chemicals used in science.

Produits et services

Chemicals for use in the pharmaceutical industry; chemical reagents for research; laboratory chemicals for scientific use; chemicals used in science.

Produits et services

Chemicals for use in the pharmaceutical industry; chemical reagents for research; laboratory chemicals for scientific use; chemicals used in science.

Produits et services

Chemicals for use in medical and veterinary analysis; chemicals for use in medical and veterinary pathology; laboratory chemicals for medical use.

Abrégé

There is provided a method for promoting dynamin ring formation and/or maintenance of dynamin rings in a cell, comprising treating the cell with an effective amount of a dynamin ring stabilizer, or a prodrug or pharmaceutically acceptable salt of the dynamin ring stabilizer. The maintenance or accumulation of dynamin ring formation has particular application in the prophylaxis or treatment of a kidney disease or condition characterized by proteinuria. A dynamin ring stabilizer can be any agent that interacts with dynamin to promote dynamin ring assembly and/or inhibit dynamin ring disassembly. There are also provided methods for prophylaxis or treatment of podocyte dysfunction and/or maintaining or inducing actin cytoskeleton formation in a cell utilizing dynamin ring stabilizers, and for screening a test agent for use as a dynamin ring stabilizer.

Classes IPC  ?

• A61K 31/35 - Composés hétérocycliques ayant l'oxygène comme seul hétéro-atome d'un cycle, p. ex. fungichromine ayant des cycles à six chaînons avec un oxygène comme seul hétéro-atome d'un cycle

Produits et services

Chemicals for use in the pharmaceutical industry; chemical reagents for research; laboratory chemicals for scientific use; chemicals used in science

Produits et services

Chemicals for use in medical and veterinary analysis, namely, medical diagnostic reagents for the analysis of body fluids and tissues; chemical reagents for use in medical, veterinary [ and plant ] pathology; [ therapeutic drugs and ] laboratory chemical reagents for medical, veterinary [ and agricultural ] use

Produits et services

Chemicals for use in the pharmaceutical industry; chemical reagents for research; laboratory chemicals for scientific use; chemicals used in science

Abrégé

The invention relates to methods and assays for the detection of active Alternative Lengthening of Telomeres (ALT) activity in cells. The methods and assays involve detecting or assaying for partially double-stranded telomeric circles wherein the presence of said circles is specific for cells comprising an active ALT mechanism. In some embodiments the methods find application in, inter alia, determining the level of ALT activity in a cell, determining the ALT status of a cancer in a subject, diagnosing and/or treating disease, determining disease status, analysis of treatment efficacy, and the identification of novel therapeutic agents.

Classes IPC  ?

• C12Q 1/68 - Procédés de mesure ou de test faisant intervenir des enzymes, des acides nucléiques ou des micro-organismesCompositions à cet effetProcédés pour préparer ces compositions faisant intervenir des acides nucléiques

Abrégé

There is provided a method for promoting dynamin ring formation and/or maintenance of dynamin rings in a cell, comprising treating the cell with an effective amount of a dynamin ring stabilizer, or a prodrug or pharmaceutically acceptable salt of the dynamin ring stabilizer. The maintenance or accumulation of dynamin ring formation has particular application in the prophylaxis or treatment of a kidney disease or condition characterized by proteinuria. A dynamin ring stabilizer can be any agent that interacts with dynamin to promote dynamin ring assembly and/or inhibit dynamin ring disassembly. There are also provided methods for prophylaxis or treatment of podocyte dysfunction and/or maintaining or inducing actin cytoskeleton formation in a cell utilizing dynamin ring stabilizers, and for screening a test agent for use as a dynamin ring stabilizer.

Classes IPC  ?

• A61K 31/275 - NitrilesIsonitriles
• A61K 31/352 - Composés hétérocycliques ayant l'oxygène comme seul hétéro-atome d'un cycle, p. ex. fungichromine ayant des cycles à six chaînons avec un oxygène comme seul hétéro-atome d'un cycle condensés avec des carbocycles, p. ex. cannabinols, méthanthéline
• A61K 31/404 - Indoles, p. ex. pindolol
• A61P 13/12 - Médicaments pour le traitement des troubles du système urinaire des reins

Abrégé

There is provided a method for promoting dynamin ring formation and/or maintenance of dynamin rings in a cell, comprising treating the cell with an effective amount of a dynamin ring stabilizer, or a prodrug or pharmaceutically acceptable salt of the dynamin ring stabilizer. The maintenance or accumulation of dynamin ring formation has particular application in the prophylaxis or treatment of a kidney disease or condition characterized by proteinuria. A dynamin ring stabilizer can be any agent that interacts with dynamin to promote dynamin ring assembly and/or inhibit dynamin ring disassembly. There are also provided methods for prophylaxis or treatment of podocyte dysfunction and/or maintaining or inducing actin cytoskeleton formation in a cell utilizing dynamin ring stabilizers, and for screening a test agent for use as a dynamin ring stabilizer.

Classes IPC  ?

• A61K 31/275 - NitrilesIsonitriles
• A61K 31/404 - Indoles, p. ex. pindolol
• A61K 31/352 - Composés hétérocycliques ayant l'oxygène comme seul hétéro-atome d'un cycle, p. ex. fungichromine ayant des cycles à six chaînons avec un oxygène comme seul hétéro-atome d'un cycle condensés avec des carbocycles, p. ex. cannabinols, méthanthéline
• A61P 13/12 - Médicaments pour le traitement des troubles du système urinaire des reins

Abrégé

Methods for inhibiting adipogenesis in a cell comprising inhibiting STC2 activity with an STC2 inhibitor are described. There is also described inhibition of adipogenesis in a subject, the inhibition of accumulation of adipose tissue in a subject, and the prophylaxis or treatment of obesity in a subject, comprising administering an effective amount of at least one STC2 inhibitor to the subject.

Classes IPC  ?

• A61K 38/00 - Préparations médicinales contenant des peptides
• A61K 31/00 - Préparations médicinales contenant des ingrédients actifs organiques
• A61P 3/00 - Médicaments pour le traitement des troubles du métabolisme
• A61P 3/04 - AnorexigènesMédicaments de l'obésité

Abrégé

The invention relates to compounds of Formula I or Ia having dynamin protein inhibitory activity. Compounds embodied by the invention have application in inhibiting cellular endocytosis and cytokinesis, and may be used in the prophylaxis or treatment of various diseases and conditions involving endocytosis or cell proliferation, such as seizures, epilepsy and cancer. Particularly preferred compounds provided by Formula I and Ia are indoles.

Classes IPC  ?

• C07D 209/00 - Composés hétérocycliques contenant des cycles à cinq chaînons condensés avec d'autres cycles, ne comportant qu'un atome d'azote comme unique hétéro-atome du cycle
• A61K 31/00 - Préparations médicinales contenant des ingrédients actifs organiques
• C07D 209/04 - IndolesIndoles hydrogénés

Abrégé

There are provided phospho-mimetics of a region of DynII including S764 or a phosphorylatable amino acid in a homologous position. The phospho-mimetics find use in inhibiting cytokinesis and have application in prophylaxis or treatment of cell proliferative diseases and conditions such as cancer. There is also provided assays for screening for inhibitors of cytokinesis.

Classes IPC  ?

• A61K 38/16 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés
• A61P 35/00 - Agents anticancéreux