RNA molecules comprising a guide sequence portion having 17-20 nucleotides in the sequence of 17-20 contiguous nucleotides set forth in any one of SEQ ID Nos: 1-1313 and compositions, methods, and uses thereof.
Compositions comprising an RNA molecule comprising a guide sequence portion having 17-50 contiguous nucleotides containing nucleotides in the sequence set forth in any one of SEQ ID NOs: 1-6838 and methods and uses thereof.
A61K 40/11 - Lymphocytes T, p. ex. lymphocytes infiltrant les tumeurs [TIL] ou lymphocytes T régulateurs [Treg]Cellules tueuses activées par les lymphokines [LAK]
Compositions comprising an RNA molecule comprising a guide sequence portion having 17-50 contiguous nucleotides containing nucleotides in the sequence set forth in any one of SEQ ID NOs: 1-34199 and methods and uses thereof.
A61K 40/50 - Immunothérapie cellulaire caractérisée par l’utilisation de cellules allogéniques
A61K 40/11 - Lymphocytes T, p. ex. lymphocytes infiltrant les tumeurs [TIL] ou lymphocytes T régulateurs [Treg]Cellules tueuses activées par les lymphokines [LAK]
Compositions comprising an RNA molecule comprising a guide sequence portion having 17-50 contiguous nucleotides containing nucleotides in the sequence set forth in any one of SEQ ID NOs: 1-4891 and methods and uses thereof.
A61K 40/11 - Lymphocytes T, p. ex. lymphocytes infiltrant les tumeurs [TIL] ou lymphocytes T régulateurs [Treg]Cellules tueuses activées par les lymphokines [LAK]
Compositions comprising an RNA molecule comprising a guide sequence portion having 17-50 contiguous nucleotides containing nucleotides in the sequence set forth in any one of SEQ ID NOs: 1-6220 and methods and uses thereof.
Methods for inactivating in a cell a mutant allele of the elastase, neutrophil expressed gene (ELANE gene) gene having a mutation associated with severe congenital neutropenia (SCN) or cyclic neutropenia (CyN) and which cell is heterozygous at one or more polymorphic sites selected from the group consisting of: rs10414837, rs3761005, rs1683564, rs9749274, rs740021, rs201048029, rs199720952, rs28591229, rs71335276, rs58082177, rs3826946, rs10413889, rs761481944, rs3761008, rs10409474, rs3761007, rs17216649, rs10469327, rs8107095, rs10424470 and rs78302854, the method comprising
introducing to the cell a composition comprising:
a CRISPR nuclease or a sequence encoding the CRISPR nuclease; and
a first RNA molecule comprising a guide sequence portion having 17-20 nucleotides,
wherein a complex of the CRISPR nuclease and the first RNA molecule affects a double strand break in the mutant allele of the ELANE gene,
the method optionally further comprising introduction of a second RNA molecule comprising a guide sequence portion capable of complexing with a CRISPR nuclease, wherein the complex of the second RNA molecule and CRISPR nuclease affects a second double strand break in the ELANE gene.
C12N 15/90 - Introduction stable d'ADN étranger dans le chromosome
A61K 35/28 - Moelle osseuseCellules souches hématopoïétiquesCellules souches mésenchymateuses de toutes origines, p. ex. cellules souches dérivées de tissu adipeux
C12N 15/11 - Fragments d'ADN ou d'ARNLeurs formes modifiées
C12Q 1/6883 - Produits d’acides nucléiques utilisés dans l’analyse d’acides nucléiques, p. ex. amorces ou sondes pour les maladies provoquées par des altérations du matériel génétique
7.
Differential Knockout of An Allele of A Heterozygous Fibrinogen Alpha Chain (FGA) Gene
RNA molecules comprising a guide sequence portion having 17-20 nucleotides in the sequence of 17-20 contiguous nucleotides set forth in any one of SEQ ID NOs: 1-1990 and compositions, methods, and uses thereof.
C12N 15/11 - Fragments d'ADN ou d'ARNLeurs formes modifiées
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
A61P 13/12 - Médicaments pour le traitement des troubles du système urinaire des reins
RNA, compositions, and methods for inactivating in a cell a mutant allele of the elastase, neutrophil expressed gene (ELANE gene) gene having a mutation associated with severe congenital neutropenia (SCN) or cyclic neutropenia (CyN) and which cell is heterozygous at the polymorphic site rs1683564, the method comprising
introducing to the cell a composition comprising:
a CRISPR nuclease or a sequence encoding the CRISPR nuclease; and
a first RNA molecule comprising a guide sequence portion having 17-30 nucleotides,
wherein a complex of the CRISPR nuclease and the first RNA molecule affects a double strand break in the mutant allele of the ELANE gene
the method optionally further comprising introduction of a second RNA molecule comprising a guide sequence portion capable of complexing with a CRISPR nuclease, wherein the complex of the second RNA molecule and CRISPR nuclease affects a second double strand break in the ELANE gene.
RNA molecules comprising a guide sequence portion having 17-50 contiguous nucleotides in the sequence set forth in any one of SEQ ID NOs: 1-159641 and compositions, methods, and uses thereof. Further, wherein a method for modifying in a cell at least one allele of the Amyloid Beta Precursor Like Protein 2 (APLP2) gene is disclosed.
A61K 35/28 - Moelle osseuseCellules souches hématopoïétiquesCellules souches mésenchymateuses de toutes origines, p. ex. cellules souches dérivées de tissu adipeux
A61K 38/17 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains
The present invention is directed to, inter alia, composition and methods for genome editing. Specifically, a non-naturally occurring OMNI-50 nuclease variant having a wild-type OMNI-50 protein sequence (SEQ ID NO: 1) comprising an amino acid substitution in at least one of the following positions: R61, Y437, R478, A493, Y545, G606, K688, L690, E695, L718, R788, Q803, L805, L844, V981, K965, and K1036.
Methods for inactivating in a cell a mutant allele of the elastase, neutrophil expressed gene (ELANE gene) gene having a mutation associated with severe congenital neutropenia (SCN) or cyclic neutropenia (CyN).
The present invention provides a non-naturally occurring composition comprising a CRISPR nuclease comprising a sequence having at least 90% identity to the amino acid sequence selected from the group consisting of SEQ ID NOs: 1, 2, 3, 4, 6, 7, or 8, or a nucleic acid molecule comprising a sequence encoding the CRISPR nuclease.
RNA molecules comprising a guide sequence portion having 17-50 contiguous nucleotides in the sequence set forth in any one of SEQ ID NOs: 1-2860 and compositions, methods, and uses thereof.
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
Methods for inactivating a mutant human guanylate cyclase 2D (GUCY2D) allele comprising delivering a gRNA having a crRNA comprising at least 17 contiguous nucleotides set forth in any one of SEQ ID NOs: 237, 238, 241, 242, 247, 248, 394, 307, 413, 414, 417, 418, or 3011, compositions thereof, and methods of preventing, treating, ameliorating or slowing the progression of cone-rod dystrophies.
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 27/00 - Médicaments pour traiter les troubles des sens
The present invention provides a non-naturally occurring composition comprising a CRISPR nuclease comprising a sequence having at least 95% identity to the amino acid sequence of SEQ ID NO: 1 or a nucleic acid molecule comprising a sequence encoding the CRISPR nuclease.
The present invention is directed to, inter alia, composition and methods for genome editing. Specifically, a non-naturally occurring OMNI-79 nuclease variant having a wild-type OMNI-79 protein sequence (SEQ ID NO: 1) comprising an amino acid substitution in at least one of the following positions: I14. S1005, and E1050.
A composition comprising a non-naturally occurring RNA molecule, the RNA molecule comprising an RNA scaffold portion, the RNA scaffold portion having the structure: crRNA repeat sequence portion-tracrRNA portion: wherein the RNA scaffold portion forms a complex with and targets an OMNI-103 CRISPR nuclease to a DNA target site having complementarity to a guide sequence portion of the RNA molecule.
RNA molecules comprising a guide sequence portion having 17-50 contiguous nucleotides in the sequence set forth in any one of SEQ ID NOs: 1-7,046 and compositions, methods, and uses thereof.
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
The invention relates to a method for adoptive cell therapy or prophylaxis comprising administering SARM1-inhibited or SARM1-inactivated cells to a subject suffering from or determined to be at risk of suffering from a cancer, infection, disease, or disorder.
The present invention provides a non-naturally occurring composition comprising a CRISPR nuclease comprising a sequence having at least 95% identity to the amino acid sequence selected from the group consisting of SEQ ID NOs: 1-108 or a nucleic acid molecule comprising a sequence encoding the CRISPR nuclease.
The present invention provides a non-naturally occurring composition comprising a CRISPR nuclease comprising a sequence having at least 95% identity to the amino acid sequence selected from the group consisting of SEQ ID NOs: 1-88 or a nucleic acid molecule comprising a sequence encoding the CRISPR nuclease.
The present invention provides a non-naturally occurring composition comprising a CRISPR nuclease comprising a sequence having at least 95% identity to the amino acid sequence selected from the group consisting of SEQ ID NOs: 1-11 and 13-39 or a nucleic acid molecule comprising a sequence encoding the CRISPR nuclease.
RNA molecules comprising a guide sequence portion having 17-25 contiguous nucleotides containing nucleotides in the sequence set forth in any one of SEQ ID NOs: 1-6601 and compostions, methods, and uses thereof.
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
A61P 37/00 - Médicaments pour le traitement des troubles immunologiques ou allergiques
The present invention disclosed herein are compositions that may be utilized for modifying genomic DNA sequences. comprising a non-naturally occurring composition comprising a CRISPR nuclease comprising a sequence having at least 95% identity to the amino acid sequence selected from the group consisting of SEQ ID NOs: 1-17 or a nucleic acid molecule comprising a sequence encoding the CRISPR nuclease.
RNA molecules comprising a guide sequence portion having 17-50 contiguous nucleotides containing nucleotides in the sequence set forth in any one of SEQ ID NOs: 1-18936 and compositions, methods, and uses thereof.
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 7/00 - Virus, p. ex. bactériophagesCompositions les contenantLeur préparation ou purification
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
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
RNA molecules comprising a guide sequence portion having 17-50 contiguous nucleotides in the sequence set forth in any one of SEQ ID NOs: 1-24195 and compositions, methods, and uses thereof.
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
A61K 39/00 - Préparations médicinales contenant des antigènes ou des anticorps
C12N 15/10 - Procédés pour l'isolement, la préparation ou la purification d'ADN ou d'ARN
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
C12N 15/90 - Introduction stable d'ADN étranger dans le chromosome
RNA molecules comprising a guide sequence portion having 17-50 contiguous nucleotides in the sequence set forth in any one of SEQ ID NOs: 1-25686 and compositions, methods, and uses thereof.
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
A61K 39/00 - Préparations médicinales contenant des antigènes ou des anticorps
C12N 15/10 - Procédés pour l'isolement, la préparation ou la purification d'ADN ou d'ARN
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
The present invention provides a composition comprising a non-naturally nuclease variant having at least 90% identity to SEQ ID NO: 1 and comprising an amino acid substitution in at least one of the following positions: N300, G614, N698, E836, T939, L1100, D252, D281, L302, N368, S1339, and S779 relative to SEQ ID NO: 1.
The present invention provides a non-naturally occurring composition comprising a CRISPR nuclease comprising a sequence having at least 95% identity to the amino acid sequence of SEQ ID NO: 3 or a nucleic acid molecule comprising a sequence encoding the CRISPR nuclease.
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
The present invention relates to compositions and methods for increasing the rate of site specific insertion of a donor DNA sequence to the genome. More specifically, the method introduces a donor DNA template containing at least one transcription factor binding site to a cell in order to favor specific insertion of a donor template sequence at a target site by homology directed repair (HDR).
The present invention provides a non-naturally occurring composition comprising a CRISPR nuclease comprising a sequence having at least 90% identity to the amino acid sequence selected from the group consisting of SEQ ID NOs: 1-22 or a nucleic acid molecule comprising a sequence encoding the CRISPR nuclease.
The present invention provides a non-naturally occurring composition comprising a CRISPR nuclease comprising a sequence having at least 90% identity to the amino acid sequence of SEQ ID NO: 1 or a nucleic acid molecule comprising a sequence encoding the CRISPR nuclease.
RNA molecules comprising a guide sequence portion having 17-50 contiguous nucleotides containing nucleotides in the sequence set forth in any one of SEQ ID NOs: 1-10736 and compositions, methods, and uses thereof.
RNA molecules comprising a guide sequence portion having 17-50 contiguous nucleotides containing nucleotides in the sequence set forth in any one of SEQ ID NOs: 1-1932 and compositions, methods, and uses thereof.
RNA molecules comprising a guide sequence portion having 17-50 contiguous nucleotides containing nucleotides in the sequence set forth in any one of SEQ ID NOs: 1-2325 and compositions, methods, and uses thereof.
RNA molecules comprising a guide sequence portion having 17-20 nucleotides in the sequence of 17-20 contiguous nucleotides set forth in any one of SEQ ID NOs: 1-3010 and compositions, methods, and uses thereof.
Compositions comprising an RNA molecule comprising a guide sequence portion having 17- 50 contiguous nucleotides containing nucleotides in the sequence set forth in any one of SEQ ID NOs: 1-37421 and methods and uses thereof.
C12N 15/74 - Vecteurs ou systèmes d'expression spécialement adaptés aux hôtes procaryotes autres que E. coli, p. ex. Lactobacillus, Micromonospora
A61K 31/711 - Acides désoxyribonucléiques naturels, c.-à-d. contenant uniquement des 2'-désoxyriboses liés à l'adénine, la guanine, la cytosine ou la thymine et ayant des liaisons 3'-5' phosphodiester
Compositions comprising an RNA molecule comprising a guide sequence portion having 17- 50 contiguous nucleotides containing nucleotides in the sequence set forth in any one of SEQ ID NOs: 1-7830 and methods and uses thereof.
Compositions comprising an RNA molecule comprising a guide sequence portion having 17- 50 contiguous nucleotides containing nucleotides in the sequence set forth in any one of SEQ ID NOs: 1-6833 and methods and uses thereof.
Compositions comprising an RNA molecule comprising a guide sequence portion having 17-50 contiguous nucleotides containing nucleotides in the sequence set forth in any one of SEQ ID NOs: 1-6215 and methods and uses thereof, or inactivating alleles of the Programmed cell death protein 1 (PDCD1) gene in a cell, such that the cell has improved activity, retention, and/or expansion qualities for use in adoptive cancer immunotherapy.
Compositions comprising an RNA molecule comprising a guide sequence portion having 17- 50 contiguous nucleotides containing nucleotides in the sequence set forth in any one of SEQ ID NOs: 1-34194 and methods and uses thereof.
A61K 31/711 - Acides désoxyribonucléiques naturels, c.-à-d. contenant uniquement des 2'-désoxyriboses liés à l'adénine, la guanine, la cytosine ou la thymine et ayant des liaisons 3'-5' phosphodiester
C12N 15/74 - Vecteurs ou systèmes d'expression spécialement adaptés aux hôtes procaryotes autres que E. coli, p. ex. Lactobacillus, Micromonospora
Compositions comprising an RNA molecule comprising a guide sequence portion having 17- 50 contiguous nucleotides containing nucleotides in the sequence set forth in any one of SEQ ID NOs: 1-4886 and methods and uses thereof.
Compositions comprising an RNA molecule comprising a guide sequence portion having 17-50 contiguous nucleotides containing nucleotides in the sequence set forth in any one of SEQ ID NOs: 1-11121 and methods and uses thereof.
RNA molecules comprising a guide sequence portion having 17-50 contiguous nucleotides in the sequence set forth in any one of SEQ ID NOs: 1-28998 and compositions, methods, and uses thereof.
A61K 35/17 - LymphocytesLymphocytes BLymphocytes TCellules tueuses naturellesLymphocytes activés par un interféron ou une cytokine
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
RNA molecules comprising a guide sequence portion having 17-50 contiguous nucleotides containing nucleotides in the sequence set forth in any one of SEQ ID NOs: 1-1538 and compositions, methods, and uses thereof.
RNA molecules comprising a guide sequence portion consisting of 17-50 nucleotides and containing 17-22 nucleotides in the sequence set forth in any one of SEQ ID NOs: 1-20347, and compositions, methods, and uses thereof.
RNA molecules comprising a guide sequence portion having 17-50 contiguous nucleotides in the sequence set forth in any one of SEQ ID NOs: 1-159641 and compositions, methods, and uses thereof. Further, wherein a method for modifying in a cell at least one allele of the Amyloid Beta Precursor Like Protein 2 (APLP2) gene is disclosed.
A composition comprising a non-naturally occurring RNA molecule, the RNA molecule comprising an RNA scaffold portion, the RNA scaffold portion having the structure:
crRNA repeat sequence portion-Linker portion-tracrRNA portion;
wherein the RNA scaffold portion forms a complex with and targets an OMNI-50 CRISPR nuclease to a DNA target site having complementarity to a guide sequence portion of the RNA molecule.
RNA molecules comprising a guide sequence portion having 17-50 contiguous nucleotides in the sequence set forth in any one of SEQ ID NOs: 1-103,606 and compositions, methods, and uses thereof.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
The present invention provides a non-naturally occurring composition comprising a CRISPR nuclease comprising a sequence having at least 90% identity to the amino acid sequence selected from the group consisting of SEQ ID NOs: 1-8 or a nucleic acid molecule comprising a sequence encoding the CRISPR nuclease.
RNA molecules comprising a guide sequence portion having 17-50 contiguous nucleotides containing nucleotides in the sequence set forth in any one of SEQ ID NOs: 1-20640 and compositions, methods, and uses thereof.
The present invention provides a non-naturally occurring composition comprising a CRISPR nuclease comprising a sequence having at least 95% identity to the amino acid sequence of SEQ ID NO: 1 or a nucleic acid molecule comprising a sequence encoding the CRISPR nuclease.
RNA molecules comprising a guide sequence portion having 17-50 contiguous nucleotides containing nucleotides in the sequence set forth in any one of SEQ ID NOs: 1-12105 and compositions, methods, and uses thereof.
RNA molecules comprising a guide sequence portion having 17-50 contiguous nucleotides containing nucleotides in the sequence set forth in any one of SEQ ID NOs: 1-20246 and compositions, methods, and uses thereof.
A composition comprising a non-naturally occurring RNA molecule, the RNA molecule comprising an RNA scaffold portion, the RNA scaffold portion having the structure: crRNA repeat sequence portion - tracrRNA portion; wherein the RNA scaffold portion forms a complex with and targets an OMNI- 103 CRISPR nuclease to a DNA target site having complementarity to a guide sequence portion of the RNA molecule.
A composition comprising a non-naturally occurring RNA molecule, the RNA molecule comprising an RNA scaffold portion, the RNA scaffold portion having the structure: crRNA repeat sequence portion - tracrRNA portion; wherein the RNA scaffold portion forms a complex with and targets an OMNI- 103 CRISPR nuclease to a DNA target site having complementarity to a guide sequence portion of the RNA molecule.
RNA molecules comprising a guide sequence portion having 17-25 contiguous nucleotides containing nucleotides in the sequence set forth in any one of SEQ ID NOs: 1-6601 and compositions, methods, and uses thereof.
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
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 37/00 - Médicaments pour le traitement des troubles immunologiques ou allergiques
RNA molecules comprising a guide sequence portion having 17-20 nucleotides in the sequence of 17-20 contiguous nucleotides set forth in any one of SEQ ID NOs: 1-3010 and compositions, methods, and uses thereof
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
RNA molecules comprising a guide sequence portion having 17-20 nucleotides in the sequence of 17-20 contiguous nucleotides set forth in any one of SEQ ID NOs: 1-3010 and compositions, methods, and uses thereof.
C12N 15/11 - Fragments d'ADN ou d'ARNLeurs formes modifiées
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 27/00 - Médicaments pour traiter les troubles des sens
RNA molecules comprising a guide sequence portion having 17-20 nucleotides in the sequence of 17-20 contiguous nucleotides set forth in any one of SEQ ID NOs: 1-3010 and compositions, methods, and uses thereof.
C12N 15/11 - Fragments d'ADN ou d'ARNLeurs formes modifiées
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 27/00 - Médicaments pour traiter les troubles des sens
RNA molecules comprising a guide sequence portion having 17-20 nucleotides in the sequence of 17-20 contiguous nucleotides set forth in any one of SEQ ID NOs: 1-3010 and compositions, methods, and uses thereof.
The invention relates to a method for adoptive cell therapy or prophylaxis comprising administering SARM1 -inhibited or SARM1 -inactivated cells to a subject suffering from or determined to be at risk of suffering from a cancer, infection, disease, or disorder.
The present invention is directed to, inter alia, composition and methods for genome editing. Specifically, a non-naturally occurring OMNI-79 nuclease variant having a wild-type OMNI-79 protein sequence (SEQ ID NO: 1) comprising an amino acid substitution in at least one of the following positions: I14, S1005, and E1050.
A61K 35/28 - Moelle osseuseCellules souches hématopoïétiquesCellules souches mésenchymateuses de toutes origines, p. ex. cellules souches dérivées de tissu adipeux
The present invention is directed to, inter alia, composition and methods for genome editing. Specifically, a non-naturally occurring OMNI-79 nuclease variant having a wild-type OMNI-79 protein sequence (SEQ ID NO: 1) comprising an amino acid substitution in at least one of the following positions: I14, S1005, and E1050.
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
A61K 35/28 - Moelle osseuseCellules souches hématopoïétiquesCellules souches mésenchymateuses de toutes origines, p. ex. cellules souches dérivées de tissu adipeux
80.
REDUCED EXPRESSION OF SARM1 FOR USE IN CELL THERAPY
The invention relates to a method for adoptive cell therapy or prophylaxis comprising administering SARM1 -inhibited or SARM1 -inactivated cells to a subject suffering from or determined to be at risk of suffering from a cancer, infection, disease, or disorder.
The present invention provides a non-naturally occurring composition comprising a CRISPR nuclease comprising a sequence having at least 95% identity to the amino acid sequence selected from the group consisting of SEQ ID NOs: 1-108 or a nucleic acid molecule comprising a sequence encoding the CRISPR nuclease.
The present invention provides a non-naturally occurring composition comprising a CRISPR nuclease comprising a sequence having at least 95% identity to the amino acid sequence selected from the group consisting of SEQ ID NOs: 1-108 or a nucleic acid molecule comprising a sequence encoding the CRISPR nuclease.
The present invention provides a non-naturally occurring composition comprising a CRISPR nuclease comprising a sequence having at least 95% identity to the amino acid sequence selected from the group consisting of SEQ ID NOs: 1-8 or a nucleic acid molecule comprising a sequence encoding the CRISPR nuclease.
The present invention is directed to, inter alia, composition and methods for genome editing. Specifically, a non-naturally occurring OMNI-50 nuclease variant having a wild-type OMNI-50 protein sequence (SEQ ID NO: 1) comprising an amino acid substitution in at least one of the following positions: R61, ¥437, R478, A493, ¥545, G606, K688, L690, E695, L718, R788, Q803, L805, L844, V981, K965, and K1036.
A61K 35/28 - Moelle osseuseCellules souches hématopoïétiquesCellules souches mésenchymateuses de toutes origines, p. ex. cellules souches dérivées de tissu adipeux
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 5/078 - Cellules du sang ou du système immunitaire
C12N 5/10 - Cellules modifiées par l'introduction de matériel génétique étranger, p. ex. cellules transformées par des virus
The present invention disclosed herein are compositions that may be utilized for modifying genomic DNA sequences, comprising a non-naturally occurring composition comprising a CRISPR nuclease comprising a sequence having at least 95% identity to the amino acid sequence selected from the group consisting of SEQ ID NOs: 1-17 or a nucleic acid molecule comprising a sequence encoding the CRISPR nuclease.
The present invention is directed to, inter alia, composition and methods for genome editing. Specifically, a non-naturally occurring OMNI-50 nuclease variant having a wild-type OMNI-50 protein sequence (SEQ ID NO: 1) comprising an amino acid substitution in at least one of the following positions: R61, ¥437, R478, A493, ¥545, G606, K688, L690, E695, L718, R788, Q803, L805, L844, V981, K965, and K1036.
The present invention disclosed herein are compositions that may be utilized for modifying genomic DNA sequences, comprising a non-naturally occurring composition comprising a CRISPR nuclease comprising a sequence having at least 95% identity to the amino acid sequence selected from the group consisting of SEQ ID NOs: 1-17 or a nucleic acid molecule comprising a sequence encoding the CRISPR nuclease.
RNA molecules comprising a guide sequence portion having 17-50 contiguous nucleotides containing nucleotides in the sequence set forth in any one of SEQ ID NOs: 1-18936 and compositions, methods, and uses thereof.
RNA molecules comprising a guide sequence portion having 17-50 contiguous nucleotides containing nucleotides in the sequence set forth in any one of SEQ ID NOs: 1-18936 and compositions, methods, and uses thereof.
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
Methods for inactivating in a cell a mutant allele of the elastase, neutrophil expressed gene (ELANE gene) gene having a mutation associated with severe congenital neutropenia (SCN) or cyclic neutropenia (CyN) and which cell is heterozygous at one or more polymorphic sites selected from the group consisting of: rs10424470, rs4807932, rs10414837, rs376107533, rs3761010, rs351108, rs3826946, rs10413889, rs3761007, rs10409474, rs3761005, rs351107, rs3761001, rs740021, rs781452480, rs371057361, rs570466264, rs1041904080, rs7250194, rs17216649, rs199720952, rs6510983, rs17223066, rs7255385, rs9749274, rs111361200, rs112639467, rs141213775, rs28591229, rs10469327, rs3834645, rs1683564, rs71335276, and rs8107095, the method comprising
introducing to the cell a composition comprising:
a CRISPR nuclease or a sequence encoding the CRISPR nuclease; and
a first RNA molecule comprising a guide sequence portion having 21-30 nucleotides,
wherein a complex of the CRISPR nuclease and the first RNA molecule affects a double strand break in the mutant allele of the ELANE gene.
The present invention provides a non-naturally occurring composition comprising a CRISPR nuclease comprising a sequence having at least 95% identity to the amino acid sequence selected from the group consisting of SEQ ID NOs: 1-88 or a nucleic acid molecule comprising a sequence encoding the CRISPR nuclease.
The present invention provides a non-naturally occurring composition comprising a CRISPR nuclease comprising a sequence having at least 95% identity to the amino acid sequence selected from the group consisting of SEQ ID NOs: 1-88 or a nucleic acid molecule comprising a sequence encoding the CRISPR nuclease.
The disclosure provides RNA molecules comprising a guide sequence portion having 17-50 contiguous nucleotides containing nucleotides in the sequence set forth in any one of SEQ ID NOs: 1-10736 and compositions, methods, and uses thereof. Specifically, the disclosure provides a method for increasing the endogenous expression of a Low-Density Lipoprotein Receptor (LDLR) gene in a cell, the method comprising modifying the LDLR gene or a transcript encoded by the LDLR gene, introducing to the cell a composition comprising at least one nuclease, and an RNA molecule comprising a guide sequence portion, wherein the guide sequence portion of the RNA molecule comprises 17-50 contiguous nucleotides containing nucleotides in the sequence set forth in any one of SEQ ID NOs: 1-10736.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
C07H 21/04 - Composés contenant au moins deux unités mononucléotide comportant chacune des groupes phosphate ou polyphosphate distincts liés aux radicaux saccharide des groupes nucléoside, p. ex. acides nucléiques avec le désoxyribosyle comme radical saccharide
C12N 5/071 - Cellules ou tissus de vertébrés, p. ex. cellules humaines ou tissus humains
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 15/87 - Introduction de matériel génétique étranger utilisant des procédés non prévus ailleurs, p. ex. co-transformation
94.
CRISPR COMPOSITIONS AND METHODS FOR PROMOTING GENE EDITING OF GATA2
RNA molecules comprising a guide sequence portion having 17-25 contiguous nucleotides containing nucleotides in the sequence set forth in any one of SEQ ID NOs: 1-44348 and compositions, methods, and uses thereof.
C12N 15/90 - Introduction stable d'ADN étranger dans le chromosome
A61K 35/28 - Moelle osseuseCellules souches hématopoïétiquesCellules souches mésenchymateuses de toutes origines, p. ex. cellules souches dérivées de tissu adipeux
C12N 15/11 - Fragments d'ADN ou d'ARNLeurs formes modifiées
The disclosure provides RNA molecules comprising a guide sequence portion having 17-50 contiguous nucleotides containing nucleotides in the sequence set forth in any one of SEQ ID NOs: 1-10736 and compositions, methods, and uses thereof. Specifically, the disclosure provides a method for increasing the endogenous expression of a Low-Density Lipoprotein Receptor (LDLR) gene in a cell, the method comprising modifying the LDLR gene or a transcript encoded by the LDLR gene, introducing to the cell a composition comprising at least one nuclease, and an RNA molecule comprising a guide sequence portion, wherein the guide sequence portion of the RNA molecule comprises 17-50 contiguous nucleotides containing nucleotides in the sequence set forth in any one of SEQ ID NOs: 1-10736.
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 5/071 - Cellules ou tissus de vertébrés, p. ex. cellules humaines ou tissus humains
C12N 15/87 - Introduction de matériel génétique étranger utilisant des procédés non prévus ailleurs, p. ex. co-transformation
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
C07H 21/04 - Composés contenant au moins deux unités mononucléotide comportant chacune des groupes phosphate ou polyphosphate distincts liés aux radicaux saccharide des groupes nucléoside, p. ex. acides nucléiques avec le désoxyribosyle comme radical saccharide
The present invention provides a novel gene editing composition comprising at least one fusion protein, which comprises a retrotransposon-encoded protein portion linked to a CRISPR nuclease portion, and an RNA template molecule comprising an insert template sequence.
Disclosure provides RNA molecules comprising a guide sequence portion having 17-50 contiguous nucleotides in the sequence set forth in any one of SEQ ID NOs: 1-7,046 and compositions, methods, and uses thereof. Specifically, the disclosure provides a method for modifying in a cell an allele of the Complement component 3 (C3) gene, the method comprising introducing to the cell a composition comprising: at least one nuclease and a RNA molecule comprising a guide sequence having 17-50 nucleotides provided.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
C07H 21/04 - Composés contenant au moins deux unités mononucléotide comportant chacune des groupes phosphate ou polyphosphate distincts liés aux radicaux saccharide des groupes nucléoside, p. ex. acides nucléiques avec le désoxyribosyle comme radical saccharide
C12N 5/071 - Cellules ou tissus de vertébrés, p. ex. cellules humaines ou tissus humains
C12N 15/87 - Introduction de matériel génétique étranger utilisant des procédés non prévus ailleurs, p. ex. co-transformation
Disclosure provides RNA molecules comprising a guide sequence portion having 17-50 contiguous nucleotides in the sequence set forth in any one of SEQ ID NOs: 1-7,046 and compositions, methods, and uses thereof. Specifically, the disclosure provides a method for modifying in a cell an allele of the Complement component 3 (C3) gene, the method comprising introducing to the cell a composition comprising: at least one nuclease and a RNA molecule comprising a guide sequence having 17-50 nucleotides provided.
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 5/071 - Cellules ou tissus de vertébrés, p. ex. cellules humaines ou tissus humains
C12N 15/87 - Introduction de matériel génétique étranger utilisant des procédés non prévus ailleurs, p. ex. co-transformation
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
C07H 21/04 - Composés contenant au moins deux unités mononucléotide comportant chacune des groupes phosphate ou polyphosphate distincts liés aux radicaux saccharide des groupes nucléoside, p. ex. acides nucléiques avec le désoxyribosyle comme radical saccharide
99.
DIFFERENTIAL KNOCKOUT OF A HETEROZYGOUS ALLELE OF RPE65
RNA molecules comprising a guide sequence portion having 17-25 contiguous nucleotides in the sequence set forth in any one of SEQ ID NOs: 1-49516 and compositions, methods, and uses thereof.
The present invention provides a non-naturally occurring composition comprising a CRISPR nuclease comprising a sequence having at least 95% identity to the amino acid sequence of SEQ ID NO: 1 or a nucleic acid molecule comprising a sequence encoding the CRISPR nuclease.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
