The present invention refers to a molecule for use in the treatment and/or prevention of DUX4- r positive leukemia wherein said molecule is selected from the group consisting of: - an inhibitor of GTF2I expression and/or function; - an inhibitor of HDAC expression and/or function; and - an inhibitor of c-Myc expression and/or function.
C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
A61K 31/166 - Amides, e.g. hydroxamic acids having aromatic rings, e.g. colchicine, atenolol, progabide having the carbon atom of a carboxamide group directly attached to the aromatic ring, e.g. procainamide, procarbazine, metoclopramide, labetalol
A61K 31/167 - Amides, e.g. hydroxamic acids having aromatic rings, e.g. colchicine, atenolol, progabide having the nitrogen atom of a carboxamide group directly attached to the aromatic ring, e.g. lidocaine, paracetamol
A61P 35/02 - Antineoplastic agents specific for leukemia
A61K 31/407 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with one nitrogen as the only ring hetero atom, e.g. sulpiride, succinimide, tolmetin, buflomedil condensed with heterocyclic ring systems, e.g. ketorolac, physostigmine
A61K 38/17 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans
A61K 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
C07C 259/06 - Compounds containing carboxyl groups, an oxygen atom of a carboxyl group being replaced by a nitrogen atom, this nitrogen atom being further bound to an oxygen atom and not being part of nitro or nitroso groups without replacement of the other oxygen atom of the carboxyl group, e.g. hydroxamic acids having carbon atoms of hydroxamic groups bound to hydrogen atoms or to acyclic carbon atoms
The present invention provides a T-cell receptor (TCR) which binds to an immunogenic peptide when presented by a major histocompatibility complex (MHC).
C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals
A61K 39/00 - Medicinal preparations containing antigens or antibodies
The present invention relates to an isolated polynucleotide comprising from 5′ to 3′: a first homology region, a nucleotide sequence encoding a RAG1 polypeptide or a RAG1 polypeptide fragment, and a second homology region for use in treating a RAG-deficient immunodeficiency.
The present invention relates to an engineered transposase comprising a transposase operably linked to a polypeptide that binds to a component of heterochromatin. The present invention further relates to an engineered transposome complex comprising an oligonucleotide and an engineered transposase according to the invention. The present invention also relates to methods and uses of the engineered transposase of the invention and engineered transposome of the invention for making a DNA sequence library or libraries and for DNA sequencing.
The present invention relates to a method for genetic manipulation of immune cells or progenitors thereof, by lipid nanoparticles delivery of a gene modifying agent to cells.
Use of one or more inhibitor(s) of senescence for increasing the survival and/or engraftment of haematopoietic cells, haematopoietic stem cells, haematopoietic progenitor cells and/or T cells in gene therapy.
The present invention concerns a system for classifying a cataract. The system comprises a head positioning module (100) suitable for receiving a head of a patient and positioning it, a source module (201) capable of emitting electromagnetic radiation, with a frequency between 400nm and 490nm, and preferably between 450nm and 470nm and more preferably equal to 460nm, to focus it on a lens of the patient and a detector module (202) suitable for acquiring the image of the lens illuminated by electromagnetic radiation. A control unit (204) configured to control the source module (201) and the detector module (202), and to process the lens image to determine the grade of cataract based on pixel values of the image. A computerized method for classifying a cataract is also described.
A61B 3/10 - Objective types, i.e. instruments for examining the eyes independent of the patients perceptions or reactions
A61B 3/117 - Objective types, i.e. instruments for examining the eyes independent of the patients perceptions or reactions for examining the anterior chamber or the anterior chamber angle, e.g. gonioscopes
A vector for central nervous system (CNS)-specific expression, wherein the vector comprises a transgene encoding interleukin 10 (IL-10), and wherein the transgene is operably linked to one or more expression control sequence.
A product comprising: (a) a vector for liver and/or splenic phagocyte-specific expression, wherein the vector comprises a transgene operably linked to one or more expression control sequence; and (b) an immune checkpoint inhibitor or a Tr1 cell inhibitor.
An enveloped viral particle producer or packaging cell, wherein the cell is modified to decrease expression of low density lipoprotein receptor (LDLR) on the surface of the cell.
Use of cyclosporin H (CsH) or a derivative thereof for increasing the efficiency of transduction of an isolated population of cells by a viral vector and/or increasing the efficiency of gene editing of an isolated population of cells when transduced by a viral vector.
An enveloped viral particle producer or packaging cell, wherein the cell is genetically engineered to decrease expression of MHC-I on the surface of the cell.
A polynucleotide comprising a nucleotide sequence encoding methyl-CpG binding-protein 2 (MeCP2) and: (a) at least one miR-124 target sequence, and/or at least one miR-31 target sequence, and/or at least one miR-338-3p target sequence; and/or (b) a nucleotide sequence encoding an inhibitor of MeCP2 expression, optionally a nucleotide sequence encoding an shRNA that has at least 90% sequence identity to SEQ ID NO: 15, 30 or 31.
A product comprising: (a) a hematopoietic stem cell (HSC), a hematopoietic progenitor cell (HPC), a myeloid/monocyte-committed progenitor cell, a macrophage or a monocyte comprising a vector, wherein the vector comprises at least one miRNA target sequence operably linked to a transgene, wherein the miRNA target sequence prevents or reduces expression of the transgene in the HSC, HPC and/or myeloid/monocyte-committed progenitor cell, wherein the transgene encodes an orthogonal cytokine; and (b) a T cell or NK cell, wherein the T cell or NK cell expresses an orthogonal receptor.
The present invention relates to novel antagonists of the Liver X Receptors (LXRs) of formula (I) and (II) which can be used alone or in combination with other anti cancer therapies, such as the immune checkpoint blockers or cell adoptive cell therapy, preferably T cell adoptive cell therapy, to treat different cancers, including melanoma, Hodgkin lymphoma, renal, lung, bladder and head and neck cancers.
C07J 9/00 - Normal steroids containing carbon, hydrogen, halogen, or oxygen, substituted in position 17 beta by a chain of more than two carbon atoms, e.g. cholane, cholestane, coprostane
C07J 31/00 - Normal steroids containing one or more sulfur atoms not belonging to a hetero ring
A61P 5/44 - GlucocorticosteroidsDrugs increasing or potentiating the activity of glucocorticosteroids
A61K 31/575 - Compounds containing cyclopenta[a]hydrophenanthrene ring systemsDerivatives thereof, e.g. steroids substituted in position 17 beta by a chain of three or more carbon atoms, e.g. cholane, cholestane, ergosterol, sitosterol
C07J 53/00 - Steroids in which the cyclopenta[a]hydrophenanthrene skeleton has been modified by condensation with carbocyclic rings or by formation of an additional ring by means of a direct link between two ring carbon atoms
16.
COMPOSITIONS AND METHODS FOR TREATING DISEASES AND DISORDERS OF THE CENTRAL NERVOUS SYSTEM
The present invention provides compositions and methods for the treatment or prevention of a neurological disease or disorder of the central nervous system (e.g., a storage disorder, lysosomal storage disorder, neurodegenerative disease, etc.) by reconstitution of brain myeloid cell and microglia upon transplantation of hematopoietic cells enriched in microglia reconstitution potential. The invention also provides compositions and methods for ablating and reconstituting microglia.
A61K 35/12 - Materials from mammalsCompositions comprising non-specified tissues or cellsCompositions comprising non-embryonic stem cellsGenetically modified cells
A01K 67/0271 - Chimeric vertebrates, e.g. comprising exogenous cells
A61K 9/00 - Medicinal preparations characterised by special physical form
A61K 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
The present invention provides a T-cell receptor (TCR) which binds to a Human Telomerase Reverse Transcriptase (hTERT) peptide or a Survivin peptide when presented by a major histocompatibility complex (MHC).
An image capturing method (100) using an image capturing apparatus (1) that comprises an imaging device (2) having an image catcher (3) for generating image data, the method (100) comprising displaying (S101), using an image reproducing device (4), images based on the image data, generating (S102) attribute data from a plurality of biological signals measured by a plurality of biological sensors (6), the attribute data being at least an information extracted by one or more biological signals through a computational processing and being indicative of attributes of a user at a time that the user is viewing the images displayed by the image reproducing device (4), generating (S103) significance data based on the attribute data, and associating the attribute data from which the significance data is generated with each displayed image, wherein the significance data are generated by comparing the attribute data with predetermined significance threshold values in order to identify a significance state for each attribute data, controlling (S104)
A combination of: (a) at least one deoxyribonucleoside (dN) or a derivative thereof and cyclosporin H (CsH) or a derivative thereof: or (b) at least one pyrimidine precursor and cyclosporin H (CsH) or a derivative thereof.
A61K 31/513 - PyrimidinesHydrogenated pyrimidines, e.g. trimethoprim having oxo groups directly attached to the heterocyclic ring, e.g. cytosine
A61K 31/7072 - Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines having oxo groups directly attached to the pyrimidine ring, e.g. cytidine, cytidylic acid having two oxo groups directly attached to the pyrimidine ring, e.g. uridine, uridylic acid, thymidine, zidovudine
A61K 35/28 - Bone marrowHaematopoietic stem cellsMesenchymal stem cells of any origin, e.g. adipose-derived stem cells
The present invention refers to a peptide or variants thereof wherein the peptide essentially consists of: - an amino acid sequence comprised in the sequence from aa. 1 to aa. 288 of matrin 3 (MATR3) (SEQ ID NO:1), or - a variant of an amino acid sequence comprised in the sequence from aa. 1 to aa. 288 of matrin 3 (MATR3), with the proviso that the peptide does not consist of the amino acid sequence from aa.1 to aa. 287 of matrin 3 (SEQ ID NO:17), and preferably wherein amino acid sequence is comprised in the sequence from aa. 1 to aa. 287 or from aa 2 to aa. 288 (SEQ ID NO:18) or from aa. 2 to aa. 287 (SEQ ID NO:19)
C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals
Method (100) for analyzing a user's reaction to at least one stimulus, wherein the method (100) comprises: providing (S101) at least one stimulus (ST) to the user selected from a plurality of stimuli according to an input information (I- Inf o ) and at least one stimulation parameter (SP) from a plurality of stimulation parameters through one or more sensory stimulators (2); measuring (S102) a biological response following the providing of said stimulus (ST) and acquiring at least one biosignal through one or more biometric sensors (3), wherein the biosignal indicates a neurophysiological reaction to the stimulus (ST) and comprises neurophysiological reaction data (RD); associating (S103) the neurophysiological reaction data (RD) with the corresponding stimulus (ST) through at least one processor (4) to generate at least one piece of sensory metadata (MD) indicative of the user's response to the corresponding stimulus (ST); archiving (S104) the sensory metadata (MD) within a memory support (5) to obtain a plurality of sensory metadata (MD) associated with the user; processing (S105) each piece of sensory metadata (MD) stored in the memory support (5) through the processor (4) according to the input information (I-Info); generating (S106) an output information (O-Info) following the processing of each piece of sensory metadata (MD), the output information (O-Info) comprising a reaction index (RI) to the stimulus as a function of the input information ( I-Info), and modifying (S107) at least one stimulation parameter (SP) according to the reaction index (RI).
A polynucleotide comprising a nucleotide sequence encoding an engineered sacsin, wherein the engineered sacsin comprises a UbL domain or variant thereof, an SRR domain or variant thereof, a DnaJ domain or variant thereof and an HERN domain or variant thereof, and wherein the nucleotide sequence encoding the engineered sacsin is less than or equal to about 4000 bp in length.
A61K 39/00 - Medicinal preparations containing antigens or antibodies
C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals
C12N 15/10 - Processes for the isolation, preparation or purification of DNA or RNA
C12N 15/11 - DNA or RNA fragmentsModified forms thereof
C12N 15/63 - Introduction of foreign genetic material using vectorsVectorsUse of hosts thereforRegulation of expression
C12N 15/85 - Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
The present invention relates to chimeric antigen receptors (CARs) that target Cadherin-17 (CDH17) and cells comprising said CARs. Methods and uses involving the CARs of the invention are also provided.
C07K 16/28 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals
C12N 5/0783 - T cellsNK cellsProgenitors of T or NK cells
The present invention is directed to a method for ex-vivo-engineering of cells, in particular stem cells or T cells, preferably hematopoietic stem and/or progenitor cells, mesenchymal stem cells, or T cells comprising a step of culturing the cells on a three-dimensional scaffold. The method of the invention is capable of improving the efficiency of genetic modification of cells and the functionality of the engineered cells.
A testing method for staining in dental restorative materials, said testing method comprising: providing a sample of dental restorative material in an initial state; subjecting said sample to at least one cyclical immersion into at least one first staining liquid and into at least one second staining liquid, controlling a periodicity and an overall duration of said cyclical immersion. A testing system for staining in dental restorative materials.
A photoacoustic agent chosen among the group consisting of a metal-based nanoparticle made of gold, silver, or hybrid gold/silver, an organic photoacoustic dye, cyanine dyes, phthalein and xanthene dyes, squaraine and croconaine dyes, tetrapyrrole, BODIPY dyes, curcumin dyes, and IRDye800 linked to a ligand of the integrin family receptors, preferably a peptide containing an integrin binding motif, and antibody or part of an antibody, a peptidomimetic or an aptamer, via a crosslinker selected from the group consisting of a crosslinker bearing amino and sulfhydryl reactive groups, bearing amino and azide/alkyne reactive groups, bearing lipoamide/lipoic acid (LA) or sulfhydryl or disulphide containing compounds.
A61K 47/69 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
C07K 7/56 - Cyclic peptides containing at least one abnormal peptide link with at least one abnormal peptide link in the ring the cyclisation not occurring through 2,4-diamino-butanoic acid
C07K 7/64 - Cyclic peptides containing only normal peptide links
29.
BIOMATERIAL COATED NANOSTRUCTURES FOR PHOTOACOUSTIC IMAGING AND PHOTOTHERMAL THERAPY OF TUMOR LESIONS
ALMA MATER STUDIORUM - UNIVERSITA' DI BOLOGNA (Italy)
Inventor
Comes Franchini, Mauro
Maturi, Mirko
Locatelli, Erica
Alfano, Massimo
Alchera, Elisa
Locatelli, Irene
Curnis, Flavio
Corti, Angelo
Abstract
Metal based nanoparticles coated with a polymer functionalized with thiol groups and 5 -NH groups, said polymer preferably being selected among thiolated chitosan, thiolated and aminated alginic acid, thiolated and aminated hyaluronic acid, or a protein preferably selected from the group consisting of albumin and gelatin, or a synthetic thiolated and aminated polymer, preferably α-thio-ω-amino polyethylene glycols; wherein said groups are linked to a ligand of the integrin family receptors, 10 preferably a peptide containing an integrin binding motif, and antibody or part of an antibody, a peptidomimetic or an aptamer, via a heterobifunctional crosslinker, the crosslinker bearing functional groups able to bind to amino groups, preferably selected from the group consisting of N-hydroxysuccinimidyl ester group (NHS ester), an isocyanate group (-NCO), an isothiocyanate group (-NCS), a Sulfo-N-15 hydroxysuccinimidyl ester group (sulfo-NHS ester), or a carboxylic acid group which is connected by activation with carbodiimide coupling agents; and/or a functional group able to bind thiol groups, preferably selected from the group consisting of: a maleimide group, a terminal vinyl group or a terminal alkyne group; and/or functional groups able to bind alkynes such as azides; and/or functional groups able 20 to bind azides, such as alkynes.
A61K 47/69 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
A61K 41/00 - Medicinal preparations obtained by treating materials with wave energy or particle radiation
An epigenetic silencer factor (ESF) comprising a transcription factor DNA-binding domain operably linked to at least one epigenetic effector domain, wherein the transcription factor is an oncogenic transcription factor or a cancer-associated transcription factor.
C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals
The invention relates to means and methods for gene therapy of lysosomal storage disorders (LSDs), preferably a LSD with skeletal involvement, based on an ex vivo gene therapy approach comprising transduction of autologous hematopoietic stem and progenitor cells (HSPCs) with viral vectors for expressing enzymes that are deficient in the disorders. The final formulation is a suspension of transduced cells in culture medium for the administration to patients affected by the LSDs, preferably preceded by a conditioning regimen.
The present invention relates to deglycosylating enzymes and modified versions thereof. The invention also provides deglycosylating enzymes alongside cell-binding molecules, such as CARs, for improving the therapeutic activity of CAR-containing cells. Methods and uses involving the deglycosylating enzymes of the invention are also provided.
Shade guide device (60) for dental restoration, comprising a core (31) of opaque composite corresponding to a dentine of a dental anatomy, the core (31) being moulded and shaped with portions with differentiated thickness (A, B, C) imitating mamelons of anterior tooth, and further comprising a buccal layer (40) of translucent enamel applied on the core (31) and forming an additional thickness on at least one buccal side of the shade guide device (60) at the portions with differentiated thickness (A, B, C). A shade guide kit. A manufacturing method of a shade guide device.
An epigenetic silencer factor (ESP), or polynucleotide encoding therefor, for use in the treatment of cancer, wherein the ESF comprises a transcription factor DNA-binding domain operably linked to at least one epigenetic effector domain, wherein the transcription factor is an oncogenic transcription factor or a cancer-associated transcription factor, wherein the cancer is selected from the group consisting of: glioma, gliobastoma, medulloblastoma, astrocytoma, neuroblastomas, ependymoma, meningioma, retinoblastoma, rhabdomyosarcoma, lung cancer, prostate cancer, breast cancer, liver cancer, pancreatic cancer (e.g. human pancreatic ductal adenocarcinoma), bladder cancer, oropharyngeal cancer, kidney cancer, colon cancer (e.g. colon adenocarcinoma), colon-rectal cancer (CRC), or a metastasis of any of the foregoing.
C07K 14/34 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from bacteria from Corynebacterium (G)
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
C12N 15/67 - General methods for enhancing the expression
C12N 15/85 - Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals
A polynucleotide comprising at least one miR-124 target sequence, and/or at least one miR- 338-3p target sequence, and/or at least one miR-31 target sequence, wherein the miRNA target sequences are operably linked to a transgene.
An agent that increases the number ofKupffer cells in a subject, or a nucleotide sequence encoding therefor, for use in a method of therapy by increasing liver immune response.
The present invention refers to An in vitro method of isolating hemogenic endothelial cells (HECs) or enriched populations of HECs, comprising isolating CD32+ cells from a population of cells derived from pluripotent stem cells.
Use of cyclosporin H (CsH) or a derivative thereof for: (a) reducing or preventing T cell exhaustion and/or loss of T cell effector functions; and/or (b) increasing T cell engraftment and/or persistence.
The present invention relates to an isolated polynucleotide comprising from 5′ to 3′: a first homology region, a splice acceptor sequence, a nucleotide sequence encoding a RAG1 polypeptide, and a second homology region for use in treating a RAG-deficient immunodeficiency.
Disclosed herein are methods and compositions for inactivating TCR genes, using zinc finger nucleases (ZFNs) comprising a zinc finger protein and a cleavage domain or cleavage half-domain in conditions able to preserve cell viability. Polynucleotides encoding ZFNs, vectors comprising polynucleotides encoding ZFNs and cells comprising polynucleotides encoding ZFNs and/or cells comprising ZFNs are also provided. Disclosed herein are also methods and compositions for expressing a functional exogenous TCR in the absence of endogenous TCR expression in T lymphocytes, including lymphocytes with a central memory phenotype. Polynucleotides encoding exogenous TCR, vectors comprising polynucleotides encoding exogenous TCR and cells comprising polynucleotides encoding exogenous TCR and/or cells comprising exogenous TCR are also provided.
An adeno-associated viral (AAV) capsid protein, wherein the capsid protein comprises: (a) a peptide comprising or consisting of the amino acid sequence PGVPGRF, or a variant thereof having up to two amino acid substitutions, additions or deletions; or (b) a peptide comprising or consisting of the amino acid sequence NGVRSVG, or a variant thereof having up to two amino acid substitutions, additions or deletions.
A method for haematopoietic stem and/or progenitor cell (HSPC) transplantation in a subject in need thereof, comprising the steps: (a) administering one or more HSPC mobiliser to the subject to mobilise the subject's endogenous HSPCs; and (b) administering a population of HSPCs to the subject.
Use of a combination of (a) cyclosporin H (CsH) or a derivative thereof, and (b) a p53 inhibitor and/or an adenoviral protein, or one or more nucleotide sequences encoding therefor, for increasing the efficiency of gene editing of an isolated population of cells when transduced by a viral vector and/or increasing the efficiency of transduction of an isolated population of cells by a viral vector.
A lentiviral vector comprising a nucleotide sequence encoding low density lipoprotein receptor (LDLR) operably linked to a promoter, optionally wherein the nucleotide sequence encoding LDLR and the promoter are in a reverse orientation in the lentiviral vector.
A product comprising two or more artificial transcription repressors (ATRs), or polynucleotides encoding therefor, selected from groups (a), (b), (c) or (d): (a) an ATR comprising a DNA-binding domain operably linked to a KRAB domain or homologue thereof; (b) an ATR comprising a DNA-binding domain operably linked to a DNMT3A, DNMT3B or DNMT1 domain or homologue thereof; (c) an ATR comprising a DNA-binding domain operably linked to a DNMT3L domain or homologue thereof; and (d) an ATR comprising a DNA-binding domain operably linked to a SETDB1 domain or homologue thereof, wherein at least two of the ATRs are selected from different groups (a), (b), (c) or (d).
C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
A61K 35/12 - Materials from mammalsCompositions comprising non-specified tissues or cellsCompositions comprising non-embryonic stem cellsGenetically modified cells
A61K 38/17 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans
A cartridge is adapted to house at least one biological sample therein, where the cartridge contains at least two overlapping layers, and the layers include at least one layer of highly hydrophobic, inert, and biocompatible material, with contact angle Θc≥90°, hydrophobic layer, and optionally, at least one layer of double-sided adhesive material, where in the absence of the at least one layer of double-sided adhesive material, the overlapping layers are connected together by chemical and/or physical bonding, where each of the overlapping layers has at least one inner hole that is pervious when the layers overlap one another, and the at least one inner hole is closed by the at least one biological sample, where loaded in the cartridge.
The present invention refers to: a) at least one peptide recognizing tumor endothelial cell markers, said peptide being preferably: - an αv-integrin ligand, preferably a ligand of αvβ3, αvβ5, αvβ8, α5β1 and/or αvβ6 integrin, more preferably said peptide comprising or consisting of a sequence comprising a RGD motif or of a CgA sequence or - a ligand of CD 13 receptor, more preferably said ligand comprising or consisting of a sequence comprising a NGR motif or functional fragments or derivatives or a biologically active variant thereof and b) saporin or functional fragments, derivatives or a biologically active variant thereof.
C12N 15/62 - DNA sequences coding for fusion proteins
C07K 14/435 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans
A61K 38/16 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof
A61K 47/64 - Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
C07K 7/08 - Linear peptides containing only normal peptide links having 12 to 20 amino acids
C07K 14/415 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from plants
48.
NON-INVASIVE VENTILATION SYSTEM FOR THE PRE-HOSPITAL MANAGEMENT OF ACUTE RESPIRATORY FAILURE
A stand-alone continuous positive airways pressure, CPAP, apparatus having a face-mask and a connected electro-mechanical device to supply air to the face-mask is disclosed. The electro-mechanical device includes a pneumatic channel for flowing air to be delivered to the face mask and a control unit for managing the air pressure of the air inside the pneumatic channel. The CPAP apparatus includes a turbine fan, located in the electro-mechanical device housing, connected to the control unit for pressurizing atmospheric air. The pneumatic channel includes an inlet portion located upstream of the turbine fan to receive atmospheric air, and an outlet portion located downstream of the turbine fan to deliver the pressurized air to the face-mask through an outlet opening. The pneumatic channel also longitudinally extends from the inlet portion to the outlet portion.
The invention provides targeting RNA, single guide RNA, tracrRNA, crisprRNA and expression vectors for use in CRISPR activation (CRISPRa) methods for the treatment of neurological disorders and diseases, in particular epilepsy and pain. In some preferred embodiments, a combinatorial gene therapy approach is used, wherein expression of multiple endogenous human genes are increased in a subject, in order to achieve a greater rescue of seizures and/or behavioural deficits, and restore physiological brain function.
The present invention relates to an inhibitor of asparagine synthase for use for the treatment of a disorder characterized by renal and/or liver cyst formation and relative pharmaceutical composition.
The present invention relates to a conjugate comprising a first peptide of sequence CNGRCG (SEQ ID NO: 1) linked to the N-terminus of a protein and a compound X linked to the N-terminus of said peptide and to related medical uses.
A polynucleotide comprising a nucleotide sequence encoding an epidermal growth factor receptor (EGFR) extracellular epitope operably linked to: (a) a NGFR or GMS SFR alpha signal peptide; (b) a EGFR or NGFR transmembrane domain; and/or (c) a NGFR or EGFR cytosplasmic tail.
C07K 16/28 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
A61P 37/06 - Immunosuppressants, e.g. drugs for graft rejection
The present invention refers to a method to produce a T cell with advantageous properties. The invention also refers to a T cell or an engineered T cell produced by the method and its use in therapy.
A method for analysing insertion sites of an exogenous nucleotide sequence in a subject's genome, wherein the method comprises: (a) providing a sample from the subject comprising cell-free double-stranded DNA polynucleotides; (b) blunting the ends of the polynucleotides; (c) ligating an oligonucleotide to both ends of the polynucleotides; (d) amplifying polynucleotides comprising an insertion site; and (e) sequencing the product of step (d).
Istituto Nazionale Genetica Molecolare - INGM (Italy)
Inventor
Di Fabio, Romano
Summa, Vincenzo
Bencheva, Leda Ivanova
De Francesco, Raffaele
Donnici, Lorena
Guidotti, Luca
Iannacone, Matteo
Prandi, Adolfo
De Matteo, Marilenia
Randazzo, Pietro
Abstract
The present invention relates to compounds that are inhibitors of hepatitis B virus (HBV). Compounds of this invention are useful alone or in combination with other agents for treating, ameliorating, preventing or curing HBV infection and related conditions. The present invention also relates to pharmaceutical compositions containing the compounds.
The present invention provides neutralizing monoclonal antibodies targeting the spike protein of Sarbecoviruses, such as Severe Acute Respiratory Syndrome-Coronavirus-2 (SARS-CoV-1 or SARS-CoV-2 like COVID-19). The monoclonal antibodies of the invention can inhibit or neutralize SARS-CoV-1 or SARS-CoV-2 activity and advantageously be used for treating, preventing or diagnosing COVID-19 infection in humans due to their significant cross-reactivity among SARS-CoV-2 variants.
The present invention refers to an inhibitor of substance P and/or of its receptor for use in the treatment and/or prevention of stem cell deficiency. Preferably the inhibitor is an NK1 antagonist. Preferably the stem cell deficiency is a corneal epithelial stem cell deficiency. The invention also refers to pharmaceutical compositions containing the inhibitor for use in the treatment and/or prevention of stem cell deficiency.
The present invention provides neutralizing monoclonal antibodies targeting the spike protein of Sarbecoviruses, such as Severe Acute Respiratory Syndrome-Coronavirus-2 (SARS-CoV-1 or SARS-CoV-2 like COVID-19). The monoclonal antibodies of the invention can inhibit or neutralize SARS-CoV-1 or SARS-CoV-2 activity and advantageously be used for treating, preventing or diagnosing COVID-19 infection in humans due to their significant cross-reactivity among SARS-CoV-2 variants.
Use of one or more inhibitor(s) of senescence for increasing the survival and/or engraftment of haematopoietic cells, haematopoietic stem cells, haematopoietic progenitor cells and/or T cells in gene therapy.
A61K 35/17 - LymphocytesB-cellsT-cellsNatural killer cellsInterferon-activated or cytokine-activated lymphocytes
A61K 31/7105 - Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
A61K 35/28 - Bone marrowHaematopoietic stem cellsMesenchymal stem cells of any origin, e.g. adipose-derived stem cells
A61K 39/395 - AntibodiesImmunoglobulinsImmune serum, e.g. antilymphocytic serum
Use of one or more inhibitor(s) of senescence for increasing the survival and/or engraftment of haematopoietic cells, haematopoietic stem cells, haematopoietic progenitor cells and/or T cells in gene therapy.
C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
C12N 5/10 - Cells modified by introduction of foreign genetic material, e.g. virus-transformed cells
C12N 15/10 - Processes for the isolation, preparation or purification of DNA or RNA
The present invention relates to an isolated polynucleotide comprising from 5' to 3': a first homology region, a nucleotide sequence encoding a RAG1 polypeptide or a RAG1 polypeptide fragment, and a second homology region for use in treating a RAG-deficient immunodeficiency.
C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
A61K 35/12 - Materials from mammalsCompositions comprising non-specified tissues or cellsCompositions comprising non-embryonic stem cellsGenetically modified cells
C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals
C12N 5/10 - Cells modified by introduction of foreign genetic material, e.g. virus-transformed cells
C12N 15/62 - DNA sequences coding for fusion proteins
C12N 15/90 - Stable introduction of foreign DNA into chromosome
62.
POLYNUCLEOTIDES USEFUL FOR CORRECTING MUTATIONS IN THE RAG1 GENE
The present invention relates to an isolated polynucleotide comprising from 5' to 3': a first homology region, a nucleotide sequence encoding a RAG1 polypeptide or a RAG1 polypeptide fragment, and a second homology region for use in treating a RAG-deficient immunodeficiency.
C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
The present invention relates to methods for the screening, diagnosis and/or prognosis of renal cell carcinoma. The methods of the invention are based on the determination of expression profiles of sets of miRNAs representative for renal cell carcinoma optionally associated with genotype analysis, in particular of uromodulin SNP variants. The invention also refers to a kit to perform such methods.
C12Q 1/6886 - Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material for cancer
The present invention refers to chromogranin A-derived peptides that are potent dual ligands for integrins αvβ6 and avβ8, their therapeutic and diagnostic uses and relative compositions.
C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals
A61K 47/64 - Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
An image capturing method (100) using an image capturing apparatus (1) that comprises an imaging device (2) having an image catcher (3) for generating image data, the method (100) comprising displaying (S101), using an image reproducing device (4), images based on the image data, generating (S102) attribute data from a plurality of biological signals measured by a plurality of biological sensors (6), the attribute data being at least an information extracted by one or more biological signals through a computational processing and being indicative of attributes of a user at a time that the user is viewing the images displayed by the image reproducing device (4), generating (S103) significance data based on the attribute data, and associating the attribute data from which the significance data is generated with each displayed image, wherein the significance data are generated by comparing the attribute data with predetermined significance threshold values in order to identify a significance state for each attribute data, controlling (S104) photographic parameters of the image capturing apparatus (1) based on the attribute data, wherein controlling the photographic parameters comprises at least a variation of said photographic parameters as a function of a variation of at least one biological signal, analysing (S105) the significance data to determine if the displayed images should be stored, wherein analysing the significance data comprises generating at least a significance level from the significance data and comparing said significance level with a triggering threshold level, and storing (S106) the image data of the displayed images together with the attribute data to a storage device (7) based on the analysed significance data.
A combination of: (a) at least one deoxyribonucleoside (dN) or a derivative thereof and cyclosporin H (CsH) or a derivative thereof; or (b) at least one pyrimidine precursor and cyclosporin H (CsH) or a derivative thereof.
A combination of: (a) at least one deoxyribonucleoside (dN) or a derivative thereof and cyclosporin H (CsH) or a derivative thereof; or (b) at least one pyrimidine precursor and cyclosporin H (CsH) or a derivative thereof.
A gene vector for use in gene therapy comprising at least one miRNA sequence target operably linked to a nucleotide sequence having a corresponding miRNA in a hematopoietic progenitor cell (HSPC) or hematopoietic stem cell (HSC) which prevents or reduces expression of the nucleotide sequence in a HSPC or HSC but not in a differentiated cell.
THE BOARD OF TRUSTEES OF THE LELAND STANFORD JUNIOR UNIVERSITY (USA)
OSPEDALE SAN RAFFAELE S.R.L. (Italy)
FONDAZIONE TELETHON (Italy)
Inventor
Bacchetta, Rosa
Roncarolo, Maria Grazia
Sato, Yohei
Naldini, Luigi
Passerini, Laura
Abstract
Engineered Treg-like cells, CD4LVFOXP3 T cells, and their use in cellular therapy to promote immune tolerance are disclosed. In particular, CD4LVFOXP3 T cells are produced by transduction of CD4+ T cells with a lentiviral vector expressing FOXP3 under the control of a constitutive promoter. Transduced cells express FOXP3 at high and persistent levels and acquire immune suppressive characteristics resembling naturally occurring Treg cells.
An epigenetic silencer factor (ESF) comprising a transcription factor DNA-binding domain operably linked to at least one epigenetic effector domain, wherein the transcription factor is an oncogenic transcription factor or a cancer-associated transcription factor.
C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals
An epigenetic silencer factor (ESF) comprising a transcription factor DNA-binding domain operably linked to at least one epigenetic effector domain, wherein the transcription factor is an oncogenic transcription factor or a cancer-associated transcription factor.
C12N 15/62 - DNA sequences coding for fusion proteins
C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals
An interleukin which binds to IL-2 receptor (IL-2R), or a nucleotide sequence encoding therefor, wherein the interleukin or nucleotide sequence is adapted to be targeted to the liver.
C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
A61K 35/17 - LymphocytesB-cellsT-cellsNatural killer cellsInterferon-activated or cytokine-activated lymphocytes
A61K 38/17 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans
The present invention relates to an engineered transposase comprising a transposase operably linked to a polypeptide that binds to a component of heterochromatin. The present invention further relates to an engineered transposome complex comprising an oligonucleotide and an engineered transposase according to the invention. The present invention also relates to methods and uses of the engineered transposase of the invention and engineered transposome of the invention for making a DNA sequence library or libraries and for DNA sequencing.
C12N 15/10 - Processes for the isolation, preparation or purification of DNA or RNA
C40B 20/04 - Identifying library members by means of a tag, label, or other readable or detectable entity associated with the library members, e.g. decoding processes
C40B 30/04 - Methods of screening libraries by measuring the ability to specifically bind a target molecule, e.g. antibody-antigen binding, receptor-ligand binding
The present invention is related to an isolated guide ribonucleic acid (gRNA) comprising a guide sequence targeting an inhibitory receptor (IR), a TCR α (TRAC) constant region or a β chain (TRBC1/2) constant region target sequence, wherein said guide sequence is selected from the group consisting of SEQ ID NOs:1-27, 122-126 and combinations thereof.
The present invention relates to a molecule for use in the treatment and/or prevention of viral infections caused by highly pathogenic coronaviruses, including Severe Acute Respiratory syndrome Coronavirus 2 (SARS-CoV-2), or variants thereof, Severe Acute Respiratory syndrome Coronavirus ((SARS)-CoV) and sarbecoviruses, said molecule being a mannose binding lectin (MBL) polypeptide, or a functional fragment, derivative, mutein or variant thereof, or an homologue having a percentage of identity with MBL polypeptide of at least 50, 60, 70, 80 or 90%, preferably for use in the treatment and/or prevention of 2019 Coronavirus disease (COVID-19).
A61K 38/17 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans
C12Q 1/6883 - Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
A61P 31/00 - Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
76.
MOLECULES FOR USE IN THE TREATMENT AND/OR PREVENTION OF COVID-19
The present invention relates to a molecule for use in the treatment and/or prevention of viral infections caused by highly pathogenic coronaviruses, including Severe Acute Respiratory syndrome Coronavirus 2 (SARS-CoV-2), or variants thereof, Severe Acute Respiratory syndrome Coronavirus ((SARS)-CoV) and sarbecoviruses, said molecule being a mannose binding lectin (MBL) polypeptide, or a functional fragment, derivative, mutein or variant thereof, or an homologue having a percentage of identity with MBL polypeptide of at least 50, 60, 70, 80 or 90%, preferably for use in the treatment and/or prevention of 2019 Coronavirus disease (COVID-19).
A61K 38/17 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans
A61P 31/00 - Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
C12Q 1/6883 - Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
77.
OXALAMIDO-SUBSTITUTED TRICYCLIC INHIBITORS OF HEPATITIS B VIRUS
ISTITUTO NAZIONALE DI GENETICA MOLECOLARE - INGM (Italy)
Inventor
De Francesco, Raffaele
Donnici, Lorena
Guidotti, Luca
Iannacone, Matteo
Di Fabio, Romano
Summa, Vincenzo
Prandi, Adolfo
Randazzo, Pietro
Ivanova Bencheva, Leda
De Matteo, Marilenia
Ferrante, Luca
Gornati, Davide
Grillo, Alessandro
Abstract
The present invention relates to compounds that are inhibitors of hepatitis B virus (HBV). Compounds of this invention are useful alone or in combination with other agents for treating, ameliorating, preventing or curing HBV infection and related conditions. The present invention also relates to pharmaceutical compositions containing the compounds.
A61K 31/407 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with one nitrogen as the only ring hetero atom, e.g. sulpiride, succinimide, tolmetin, buflomedil condensed with heterocyclic ring systems, e.g. ketorolac, physostigmine
A61K 31/522 - Purines, e.g. adenine having oxo groups directly attached to the heterocyclic ring, e.g. hypoxanthine, guanine, acyclovir
A61K 31/513 - PyrimidinesHydrogenated pyrimidines, e.g. trimethoprim having oxo groups directly attached to the heterocyclic ring, e.g. cytosine
A61K 31/7072 - Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines having oxo groups directly attached to the pyrimidine ring, e.g. cytidine, cytidylic acid having two oxo groups directly attached to the pyrimidine ring, e.g. uridine, uridylic acid, thymidine, zidovudine
A61K 31/675 - Phosphorus compounds having nitrogen as a ring hetero atom, e.g. pyridoxal phosphate
A61K 31/437 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems the heterocyclic ring system containing a five-membered ring having nitrogen as a ring hetero atom, e.g. indolizine, beta-carboline
A61K 31/7084 - Compounds having two nucleosides or nucleotides, e.g. nicotinamide-adenine dinucleotide, flavine-adenine dinucleotide
A61K 31/506 - PyrimidinesHydrogenated pyrimidines, e.g. trimethoprim not condensed and containing further heterocyclic rings
A61K 31/445 - Non-condensed piperidines, e.g. piperocaine
The present invention relates to novel aminoguanidine hydrazone-derivatives of Formula (I) which are effective as retromer stabilizers and useful as neuroprotecting drugs. The invention also relates to pharmaceutical compositions comprising the compounds and their use in therapy and diagnostic.
The present invention relates to novel aminoguanidine hydrazone-derivatives of Formula (I) which are effective as retromer stabilizers and useful as neuroprotecting drugs. The invention also relates to pharmaceutical compositions comprising the compounds and their use in therapy and diagnostic.
C07C 281/18 - Compounds containing any of the groups e.g. aminoguanidine the other nitrogen atom being further doubly-bound to a carbon atom, e.g. guanylhydrazones
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
C07C 277/08 - Preparation of guanidine or its derivatives, i.e. compounds containing the group the singly-bound nitrogen atoms not being part of nitro or nitroso groups of substituted guanidines
C07D 295/192 - Radicals derived from carboxylic acids from aromatic carboxylic acids
C07C 311/08 - Sulfonamides having sulfur atoms of sulfonamide groups bound to acyclic carbon atoms of an acyclic saturated carbon skeleton having the nitrogen atom of at least one of the sulfonamide groups bound to a carbon atom of a six-membered aromatic ring
C07C 311/21 - Sulfonamides having sulfur atoms of sulfonamide groups bound to carbon atoms of six-membered aromatic rings having the nitrogen atom of at least one of the sulfonamide groups bound to a carbon atom of a six-membered aromatic ring
A vector for liver and/or splenic phagocyte-specific expression, wherein the vector comprises a transgene operably linked to one or more expression control sequence.
A vector for liver and/or splenic phagocyte-specific expression, wherein the vector comprises a transgene operably linked to one or more expression control sequence.
An agent that increases the number of Kupffer cells in a subject, or a nucleotide sequence encoding therefor, for use in a method of therapy by increasing liver immune response.
A61K 45/00 - Medicinal preparations containing active ingredients not provided for in groups
A61K 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
A61P 1/16 - Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
A polynucleotide comprising a nucleotide sequence encoding methyl-CpG binding-protein 2 (MeCP2) operably linked to a strong promoter and a 3′-UTR, wherein the 3′-UTR is less than or equal to about 1000 bp in length.
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals
The present invention relates to an isolated polynucleotide comprising from 5' to 3': a first homology region, a splice acceptor sequence, a nucleotide sequence encoding a RAG1 polypeptide, and a second homology region for use in treating a RAG-deficient immunodeficiency.
A61K 38/17 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans
C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals
The present invention relates to an isolated polynucleotide comprising from 5' to 3': a first homology region, a splice acceptor sequence, a nucleotide sequence encoding a RAG1 polypeptide, and a second homology region for use in treating a RAG-deficient immunodeficiency.
C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals
A61K 38/17 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans
ISTITUTO NAZIONALE DI GENETICA MOLECOLARE - INGM (Italy)
Inventor
De Francesco, Raffaele
Donnici, Lorena
Guidotti, Luca
Iannacone, Matteo
Di Fabio, Romano
Summa, Vincenzo
Prandi, Adolfo
Randazzo, Pietro
Gornati, Davide
Grillo, Alessandro
Ferrante, Luca
Bencheva, Leda Ivanova
De Matteo, Marilenia
Ferrara, Marco
Abstract
The present invention relates to compounds that are inhibitors of hepatitis B virus (HBV). Compounds of this invention are useful alone or in combination with other agents for treating, ameliorating, preventing or curing HBV infection and related conditions. The present invention also relates to pharmaceutical compositions containing the compounds.
C07D 519/00 - Heterocyclic compounds containing more than one system of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring system not provided for in groups or
A61K 31/554 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having seven-membered rings, e.g. azelastine, pentylenetetrazole having at least one nitrogen and at least one sulfur as ring hetero atoms, e.g. clothiapine, diltiazem
A61K 31/407 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with one nitrogen as the only ring hetero atom, e.g. sulpiride, succinimide, tolmetin, buflomedil condensed with heterocyclic ring systems, e.g. ketorolac, physostigmine
The present invention relates to an inhibitor of DUX4 and its use, in particular in the prevention and/or treatment of a condition associated with an aberrant expression and/or function of at least one DUX4 protein and/or of at least one DUX4 fusion protein. Preferably the inhibitor is MATRIN-3 (MATR3), fragment, variant, fusion, or conjugate thereof. The invention also relates to a pharmaceutical composition comprising such inhibitor, to vector and nucleic acids.
C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals
C12N 15/63 - Introduction of foreign genetic material using vectorsVectorsUse of hosts thereforRegulation of expression
A61P 35/02 - Antineoplastic agents specific for leukemia
A61P 25/00 - Drugs for disorders of the nervous system
The present invention relates to a cartridge (6) adapted to house at least one biological sample (58) therein, wherein said cartridge comprises at least two overlapping layers (2, 3, 4, 5), wherein said layers consist of: - at least one layer consisting of highly hydrophobic, inert, and biocompatible material, with contact angle Tc= 90°, hydrophobic layer (2, 5); - optionally, at least one layer (3, 4) of double-sided adhesive material; wherein, in the absence of said at least one layer (3, 4) of double-sided adhesive material, said overlapping layers are connected together by chemical and/or physical bonding; wherein each of said overlapping layers (2, 3, 4, 5) has at least one inner hole (7) and said at least one inner hole (7) is pervious when said layers (2,3, 4, 5) overlap one another; wherein said at least one inner hole (7) is closed by said at least one biological sample (58), where loaded in said cartridge (6). The present invention further relates to a fluidic device (200, 500) and a fluidic devices-support station complex (600) comprising said cartridge, and to the use thereof in static and/or dynamic bicompartmental biological cultures.
cc≥ 90°, hydrophobic layer (2, 5); - optionally, at least one layer (3, 4) of double-sided adhesive material; wherein, in the absence of said at least one layer (3, 4) of double-sided adhesive material, said overlapping layers are connected together by chemical and/or physical bonding; wherein each of said overlapping layers (2, 3, 4, 5) has at least one inner hole (7) and said at least one inner hole (7) is pervious when said layers (2,3, 4, 5) overlap one another; wherein said at least one inner hole (7) is closed by said at least one biological sample (58), where loaded in said cartridge (6). The present invention further relates to a fluidic device (200, 500) and a fluidic devices-support station complex (600) comprising said cartridge, and to the use thereof in static and/or dynamic bicompartmental biological cultures.
A method for selecting genome edited cells and/or for enrichment of genome edited cells in a population of cells comprising: (a) introducing into a cell or a population of cells at least one first component, at least one second component and at least one third component; and (b) selecting the genome edited cells which transiently express or transiently upregulate a nucleotide sequence encoding a selector.
The present invention relates to the field of bacterial strain to be used in medicine. In particular, it relates to the prevention and/or treatment of cancer or diabetes, bacterial strain is Prevotella melaninogenica.
The present disclosure provides methods for generating hematopoietic progenitor cells. In some embodiments, the methods involve an in vitro or ex vivo cell culture model utilizing retinoic acid signaling for producing hematopoietic progenitor cells from pluripotent stem cells.
The present invention relates to an antibody or antigen binding fragment thereof that binds specifically to IGFBP3 and does not displace the binding of IGF-I to IGFBP3. The antibody inhibits or reduces the binding of IGFBP3 to the TMEM219 receptor. The invention also relates to methods for their production, pharmaceutical compositions containing said antibodies, and uses thereof.
A polynucleotide comprising a nucleotide sequence encoding an epidermal growth factor receptor (EGFR) extracellular epitope operably linked to: (a) a NGFR or GMS SFR alpha signal peptide; (b) a EGFR or NGFR transmembrane domain; and/or (c) a NGFR or EGFR cytosplasmic tail.
C07K 14/71 - ReceptorsCell surface antigensCell surface determinants for growth factorsReceptorsCell surface antigensCell surface determinants for growth regulators
C07K 16/28 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
98.
A MODIFIED EPIDERMAL GROWTH FACTOR RECEPTOR AND ITS USE IN TRACKING CELLS
A polynucleotide comprising a nucleotide sequence encoding an epidermal growth factor receptor (EGFR) extracellular epitope operably linked to: (a) a NGFR or GMS SFR alpha signal peptide; (b) a EGFR or NGFR transmembrane domain; and/or (c) a NGFR or EGFR cytosplasmic tail.
C07K 16/28 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
99.
NON-INVASIVE VENTILATION SYSTEM FOR THE PRE-HOSPITAL MANAGEMENT OF ACUTE RESPIRATORY FAILURE
Stand-alone continuous positive airways pressure, CPAP, apparatus (1) to contrast respiratory failure of a patient comprising a face-mask (10) to deliver continuous pressured air to the patient's airways, and an electro-mechanical device (20) having a housing (22) directly and rigidly connectable to the face-mask (10) to supply air to said face-mask (10) at a controlled pressure value, the electro-mechanical device (20) comprising a pneumatic channel (24) for flowing air to be delivered to the face mask (10) and a control unit (26) for automatically managing the value of the pressure of the air inside the pneumatic channel (24), wherein the apparatus (1) comprises a turbine fan (28) connected to the control unit (26) and located in the housing (22) of the electro-mechanical device (20) for pressurizing atmospheric air to a determined pressure level value, and wherein the pneumatic channel (24) includes an inlet portion (241) located upstream of the turbine fan (28) to receive atmospheric air, and an outlet portion (242) located downstream of the turbine fan (28) to deliver the pressurized air to the face-mask (10) through an outlet opening (21), the pneumatic channel (24) longitudinally extending from said inlet portion (241) to said outlet portion (242).
Stand-alone continuous positive airways pressure, CPAP, apparatus (1) to contrast respiratory failure of a patient comprising a face-mask (10) to deliver continuous pressured air to the patient's airways, and an electro-mechanical device (20) having a housing (22) directly and rigidly connectable to the face-mask (10) to supply air to said face-mask (10) at a controlled pressure value, the electro-mechanical device (20) comprising a pneumatic channel (24) for flowing air to be delivered to the face mask (10) and a control unit (26) for automatically managing the value of the pressure of the air inside the pneumatic channel (24), wherein the apparatus (1) comprises a turbine fan (28) connected to the control unit (26) and located in the housing (22) of the electro-mechanical device (20) for pressurizing atmospheric air to a determined pressure level value, and wherein the pneumatic channel (24) includes an inlet portion (241) located upstream of the turbine fan (28) to receive atmospheric air, and an outlet portion (242) located downstream of the turbine fan (28) to deliver the pressurized air to the face-mask (10) through an outlet opening (21), the pneumatic channel (24) longitudinally extending from said inlet portion (241) to said outlet portion (242).
