Use of virus, viral particle or viral elements as a vector

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C12N2750/14141

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CCHEMISTRY METALLURGY C12Biochemistry C12NMICRO-ORGANISMS OR ENZYMES COMPOSITIONS THEREOF PROPAGATING, PRESERVING OR MAINTAINING MICRO-ORGANISMS MUTATION OR GENETIC ENGINEERING CULTURE MEDIA C12N2750/00ssDNA Viruses

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C12N2750/14141Use of virus, viral particle or viral elements as a vector

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Patents Grantslast 30 patents

Information Patent Grant
Reducing abnormal accumulation of TDP-43 in motor neurons in amyotr...
- Patent number 12,161,728
- Issue date Dec 10, 2024
- Macquarie University
- Stephanie Rayner
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
CRISPR therapy
- Patent number 12,152,242
- Issue date Nov 26, 2024
- The Curators of the University of Missouri
- Dongsheng Duan
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Methods of predicting ancestral virus sequences and uses thereof
- Patent number 12,134,786
- Issue date Nov 5, 2024
- Schepens Eye Research Institute, Inc.
- Luk H. Vandenberghe
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Treatment of brain cancers using central nervous system mediated ge...
- Patent number 12,122,844
- Issue date Oct 22, 2024
- Cornell University
- Ronald G. Crystal
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Butyrylcholinesterases having an enhanced ability to hydrolyze acyl...
- Patent number 12,116,602
- Issue date Oct 15, 2024
- Mayo Foundation for Medical Education and Research
- Liyi Geng
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Gene therapy
- Patent number 12,059,478
- Issue date Aug 13, 2024
- Ospedale San Raffaele S.r.l.
- Anna Christina Kajaste-Rudnitski
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Methods and compositions for dual glycan binding AAV vectors
- Patent number 12,049,478
- Issue date Jul 30, 2024
- The University of North Carolina at Chapel Hill
- Aravind Asokan
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Oxidation-resistant AAT gene therapy
- Patent number 12,031,144
- Issue date Jul 9, 2024
- Cornell University
- Ronald G. Crystal
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Treatment of brain cancers using central nervous system mediated ge...
- Patent number 12,024,568
- Issue date Jul 2, 2024
- Cornell University
- Ronald G. Crystal
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Treatment of muscular dystrophies
- Patent number 12,012,439
- Issue date Jun 18, 2024
- Royal Holloway and Bedford New College
- John George Dickson
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Compositions and methods for regulating production of a precursor p...
- Patent number 11,873,505
- Issue date Jan 16, 2024
- Wyvern Pharmaceuticals Inc.
- Bradley G. Thompson
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Bicistronic AAV vector for RNA interference in ALS
- Patent number 11,872,290
- Issue date Jan 16, 2024
- Ecole Polytechnique Federale de Lausanne (EPFL)
- Julianne Aebischer
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Cas-ready mouse embryonic stem cells and mice and uses thereof
- Patent number 11,866,794
- Issue date Jan 9, 2024
- REGENERON PHARMACEUTICALS, INC.
- Guochun Gong
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Transgenic RPE cells overexpressing OTX2 for the treatment of retin...
- Patent number 11,865,189
- Issue date Jan 9, 2024
- SORBONNE UNIVERSITE
- Thierry Leveillard
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Genetic modification of the AAV capsid resulting in altered tropism...
- Patent number 11,821,009
- Issue date Nov 21, 2023
- Cornell University
- Ronald G. Crystal
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Liver targeting adeno-associated viral vectors
- Patent number 11,821,008
- Issue date Nov 21, 2023
- Biomarin Pharmaceutical Inc.
- Peter Cameron Colosi
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Adeno-associated variants, formulations and methods for pulmonary d...
- Patent number 11,807,868
- Issue date Nov 7, 2023
- 4D MOLECULAR THERAPEUTICS INC.
- Melissa Kotterman
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
CRISPR/Cas 9-mediated integration of polynucleotides by sequential...
- Patent number 11,773,409
- Issue date Oct 3, 2023
- The Board of Trustees of the Leland Stanford Junior University
- Rasmus O. Bak
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
AAV treatment of Huntington's disease
- Patent number 11,773,392
- Issue date Oct 3, 2023
- University of Massachusetts
- Christian Mueller
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Delivery of card protein as therapy for ocular inflammation
- Patent number 11,739,130
- Issue date Aug 29, 2023
- University of Florida Research Foundation, Incorporated
- Cristhian J. Ildefonso
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Adeno-associated virus vector delivery of micro-dystrophin to treat...
- Patent number 11,723,986
- Issue date Aug 15, 2023
- Research Institute at Nationwide Children's Hospital
- Louise Rodino-Klapac
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Treatment of muscular dystrophies
- Patent number 11,725,032
- Issue date Aug 15, 2023
- Royal Holloway and Bedford New College
- George Dickson
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Viral vectors encoding recombinant FVIII variants with increased ex...
- Patent number 11,707,536
- Issue date Jul 25, 2023
- Takeda Pharmaceutical Company Limited
- Hanspeter Rottensteiner
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Adeno-associated virus Factor VIII vectors, associated viral partic...
- Patent number 11,690,898
- Issue date Jul 4, 2023
- Biomarin Pharmaceutical Inc.
- Stuart Bunting
- G01 - MEASURING TESTING
Information Patent Grant
Adeno-associated virus (AAV) vector having hybrid HGF gene introduc...
- Patent number 11,634,728
- Issue date Apr 25, 2023
- Helixmith Co., Ltd.
- Seung Shin Yu
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Methods and compositions for attenuating anti-viral transfer vector...
- Patent number 11,633,422
- Issue date Apr 25, 2023
- Selecta Biosciences, Inc.
- Takashi Kei Kishimoto
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Adeno-associated viral (AAV) vectors useful for transducing adipose...
- Patent number 11,629,361
- Issue date Apr 18, 2023
- Universitat Autonoma de Barcelona
- Verónica Jiménez Cenzano
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Method of making and using mammalian liver cells for treating hemop...
- Patent number 11,591,622
- Issue date Feb 28, 2023
- Sangamo Therapeutics, Inc.
- Michael C. Holmes
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Silencing of DUX4 by recombinant gene editing complexes
- Patent number 11,566,237
- Issue date Jan 31, 2023
- University of Massachusetts
- Peter L. Jones
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Recombinant AAV variants and uses thereof
- Patent number 11,542,525
- Issue date Jan 3, 2023
- University of Massachusetts
- Guangping Gao
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE

Patents Applicationslast 30 patents

Information Patent Application
AAV CAPSID VARIANTS AND USES THEREOF
- Publication number 20240374756
- Publication date Nov 14, 2024
- VOYAGER THERAPEUTICS, INC.
- Mathieu Emmanuel Nonnenmacher
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
METHODS AND COMPOSITIONS FOR DUAL GLYCAN BINDING AAV VECTORS
- Publication number 20240327468
- Publication date Oct 3, 2024
- The University of North Carolina at Chapel Hill
- Aravind ASOKAN
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
ADENO-ASSOCIATED VIRUS (AAV) SYSTEMS FOR TREATMENT OF PROGRANULIN A...
- Publication number 20240318199
- Publication date Sep 26, 2024
- Beacon Therapeutics Limited
- Juan Li
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
COMPOSITIONS AND METHODS FOR THE TARGETING OF C9orf72
- Publication number 20240309344
- Publication date Sep 19, 2024
- Scribe Therapeutics Inc.
- Benjamin OAKES
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
SELF-POLARIZING IMMUNE CELLS
- Publication number 20240299543
- Publication date Sep 12, 2024
- Carisma Therapeutics Inc.
- Michael Klichinsky
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
ENGINEERED MEGANUCLEASES SPECIFIC FOR RECOGNITION SEQUENCES IN THE...
- Publication number 20240271111
- Publication date Aug 15, 2024
- PRECISION BIOSCIENCES, INC.
- Derek JANTZ
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
DELIVERY OF CARD PROTEIN AS THERAPY FOR OCCULAR INFLAMMATION
- Publication number 20240254178
- Publication date Aug 1, 2024
- University of Florida Research Foundation, Incorporated
- Cristhian J. Ildefonso
- C07 - ORGANIC CHEMISTRY
Information Patent Application
CONSTRUCTS AND METHODS FOR DELIVERYING MOLECULES VIA VIRAL VECTORS...
- Publication number 20240229068
- Publication date Jul 11, 2024
- NXGEN VECTOR SOLUTIONS, LLC
- Joseph E. Rabinowitz
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
MICRORNA COMPOSITIONS AND METHODS OF USE THEREOF FOR THE TREATMENT...
- Publication number 20240229031
- Publication date Jul 11, 2024
- Rutgers, The State University of New Jersey
- M. Maral Mouradian
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
CONSTRUCTS AND METHODS FOR DELIVERYING MOLECULES VIA VIRAL VECTORS...
- Publication number 20240132912
- Publication date Apr 25, 2024
- NXGEN VECTOR SOLUTIONS, LLC
- Joseph E. Rabinowitz
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
MICRORNA COMPOSITIONS AND METHODS OF USE THEREOF FOR THE TREATMENT...
- Publication number 20240132891
- Publication date Apr 25, 2024
- Rutgers, The State University of New Jersey
- M. Maral Mouradian
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
CAS-READY MOUSE EMBRYONIC STEM CELLS AND MICE AND USES THEREOF
- Publication number 20240093316
- Publication date Mar 21, 2024
- Regeneron Pharmaceuticals, Inc.
- Guochun Gong
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
AAV TREATMENT OF HUNTINGTON’S DISEASE
- Publication number 20240084303
- Publication date Mar 14, 2024
- University of Massachusetts
- Christian Mueller
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
ADENO-ASSOCIATED VARIANTS, FORMULATIONS AND METHODS FOR PULMONARY D...
- Publication number 20240067987
- Publication date Feb 29, 2024
- 4D MOLECULAR THERAPEUTICS INC.
- Melissa Kotterman
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
ADENO-ASSOCIATED VIRUS CAPSIDS AND VECTORS
- Publication number 20240067678
- Publication date Feb 29, 2024
- CHILDREN'S MEDICAL RESEARCH INSTITUTE
- Adrian WESTHAUS
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
Adeno-Associated Virus Factor VIII Vectors, Associated Viral Partic...
- Publication number 20240058423
- Publication date Feb 22, 2024
- BioMarin Pharmaceutical Inc.
- Stuart Bunting
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
METHOD AND COMPOSITION FOR TREATING NON-ALCOHOLIC FATTY LIVER DISEA...
- Publication number 20240060072
- Publication date Feb 22, 2024
- The Hong Kong Polytechnic University
- Chi Ming WONG
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
ASSAY FOR MEASURING POTENCY OF GENE THERAPY DRUG PRODUCT
- Publication number 20240044869
- Publication date Feb 8, 2024
- Prevail Therapeutics, Inc.
- Mary NG
- G01 - MEASURING TESTING
Information Patent Application
Compositions and Methods for Treating Spinal Muscular Atrophy
- Publication number 20240000886
- Publication date Jan 4, 2024
- Genzyme Corporation
- Marco A. PASSINI
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
Treatment of Muscular Dystrophies
- Publication number 20230391840
- Publication date Dec 7, 2023
- ROYAL HOLLOWAY AND BEDFORD NEW COLLEGE
- John George Dickson
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
ABCD1 FOR TREATMENT OF NEURODISORDERS
- Publication number 20230365967
- Publication date Nov 16, 2023
- The General Hospital Corporation
- Casey A. Maguire
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
IN UTERO AND POSTNATAL GENE EDITING AND THERAPY FOR TREATMENT OF MO...
- Publication number 20230340486
- Publication date Oct 26, 2023
- The Children's Hospital of Philadelphia
- William H. Peranteau
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
METHODS AND COMPOSITIONS FOR THE PRODUCTION OF ADENO-ASSOCIATED VIRUS
- Publication number 20230323395
- Publication date Oct 12, 2023
- HOMOLOGY MEDICINES, INC.
- Marissa Stanvick
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
PLASMID SYSTEM
- Publication number 20230323387
- Publication date Oct 12, 2023
- ASCEND GENE AND CELL THERAPIES LTD
- Florian Sonntag
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
METHODS OF PREDICTING ANCESTRAL VIRUS SEQUENCES AND USES THEREOF
- Publication number 20230287359
- Publication date Sep 14, 2023
- Massachusetts Eye and Ear Infirmary
- Luk H. Vandenberghe
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
NOVEL CRISPR ENZYMES, METHODS, SYSTEMS AND USES THEREOF
- Publication number 20230279373
- Publication date Sep 7, 2023
- Beam Therapeutics Inc.
- Bernd ZETSCHE
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
THERAPEUTICS FOR THE TREATMENT OF FSHD
- Publication number 20230277538
- Publication date Sep 7, 2023
- UCL Business LTD
- Julie DUMONCEAUX
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
METHODS AND COMPOSITIONS FOR THE TREATMENT OF ALS
- Publication number 20230279434
- Publication date Sep 7, 2023
- University of Cincinnati
- Alexander John MacLennan
- C07 - ORGANIC CHEMISTRY
Information Patent Application
Treatment of Genetic Dilated Cardiomyopathies
- Publication number 20230265427
- Publication date Aug 24, 2023
- GENETHON
- Isabelle Richard
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
GENE CONSTRUCTS FOR SILENCING ANGIOPOIETIN-LIKE 3 (ANGPTL3) AND USE...
- Publication number 20230265434
- Publication date Aug 24, 2023
- uniQure IP B.V.
- Sander Jan Hendrik VAN DEVENTER
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE

Use of virus, viral particle or viral elements as a vector

Industry

Parent Industries

Current Industry

Patents Grantslast 30 patents

Reducing abnormal accumulation of TDP-43 in motor neurons in amyotr...

CRISPR therapy

Methods of predicting ancestral virus sequences and uses thereof

Treatment of brain cancers using central nervous system mediated ge...

Butyrylcholinesterases having an enhanced ability to hydrolyze acyl...

Gene therapy

Methods and compositions for dual glycan binding AAV vectors

Oxidation-resistant AAT gene therapy

Treatment of brain cancers using central nervous system mediated ge...

Treatment of muscular dystrophies

Compositions and methods for regulating production of a precursor p...

Bicistronic AAV vector for RNA interference in ALS

Cas-ready mouse embryonic stem cells and mice and uses thereof

Transgenic RPE cells overexpressing OTX2 for the treatment of retin...

Genetic modification of the AAV capsid resulting in altered tropism...

Liver targeting adeno-associated viral vectors

Adeno-associated variants, formulations and methods for pulmonary d...

CRISPR/Cas 9-mediated integration of polynucleotides by sequential...

AAV treatment of Huntington's disease

Delivery of card protein as therapy for ocular inflammation

Adeno-associated virus vector delivery of micro-dystrophin to treat...

Treatment of muscular dystrophies

Viral vectors encoding recombinant FVIII variants with increased ex...

Adeno-associated virus Factor VIII vectors, associated viral partic...

Adeno-associated virus (AAV) vector having hybrid HGF gene introduc...

Methods and compositions for attenuating anti-viral transfer vector...

Adeno-associated viral (AAV) vectors useful for transducing adipose...

Method of making and using mammalian liver cells for treating hemop...

Silencing of DUX4 by recombinant gene editing complexes

Recombinant AAV variants and uses thereof

Patents Applicationslast 30 patents

AAV CAPSID VARIANTS AND USES THEREOF

METHODS AND COMPOSITIONS FOR DUAL GLYCAN BINDING AAV VECTORS

ADENO-ASSOCIATED VIRUS (AAV) SYSTEMS FOR TREATMENT OF PROGRANULIN A...

COMPOSITIONS AND METHODS FOR THE TARGETING OF C9orf72

SELF-POLARIZING IMMUNE CELLS

ENGINEERED MEGANUCLEASES SPECIFIC FOR RECOGNITION SEQUENCES IN THE...

DELIVERY OF CARD PROTEIN AS THERAPY FOR OCCULAR INFLAMMATION

CONSTRUCTS AND METHODS FOR DELIVERYING MOLECULES VIA VIRAL VECTORS...

MICRORNA COMPOSITIONS AND METHODS OF USE THEREOF FOR THE TREATMENT...

CONSTRUCTS AND METHODS FOR DELIVERYING MOLECULES VIA VIRAL VECTORS...

MICRORNA COMPOSITIONS AND METHODS OF USE THEREOF FOR THE TREATMENT...

CAS-READY MOUSE EMBRYONIC STEM CELLS AND MICE AND USES THEREOF

AAV TREATMENT OF HUNTINGTON’S DISEASE

ADENO-ASSOCIATED VARIANTS, FORMULATIONS AND METHODS FOR PULMONARY D...

ADENO-ASSOCIATED VIRUS CAPSIDS AND VECTORS

Adeno-Associated Virus Factor VIII Vectors, Associated Viral Partic...

METHOD AND COMPOSITION FOR TREATING NON-ALCOHOLIC FATTY LIVER DISEA...

ASSAY FOR MEASURING POTENCY OF GENE THERAPY DRUG PRODUCT

Compositions and Methods for Treating Spinal Muscular Atrophy

Treatment of Muscular Dystrophies

ABCD1 FOR TREATMENT OF NEURODISORDERS

IN UTERO AND POSTNATAL GENE EDITING AND THERAPY FOR TREATMENT OF MO...

METHODS AND COMPOSITIONS FOR THE PRODUCTION OF ADENO-ASSOCIATED VIRUS

PLASMID SYSTEM

METHODS OF PREDICTING ANCESTRAL VIRUS SEQUENCES AND USES THEREOF

NOVEL CRISPR ENZYMES, METHODS, SYSTEMS AND USES THEREOF

THERAPEUTICS FOR THE TREATMENT OF FSHD

METHODS AND COMPOSITIONS FOR THE TREATMENT OF ALS

Treatment of Genetic Dilated Cardiomyopathies

GENE CONSTRUCTS FOR SILENCING ANGIOPOIETIN-LIKE 3 (ANGPTL3) AND USE...