Nucleic acids adapted for tissue specific expression

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A61K48/0058

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AHUMAN NECESSITIES A61Medical science A61KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases Gene therapy

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A61K48/0058Nucleic acids adapted for tissue specific expression

Industries

Patents Grantslast 30 patents

Information Patent Grant
Compositions and processes for targeted delivery, expression and mo...
- Patent number 12,195,746
- Issue date Jan 14, 2025
- Combined Therapeutics, Inc.
- Romain Micol
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Materials and methods for treatment of hereditary haemochromatosis
- Patent number 12,186,406
- Issue date Jan 7, 2025
- CRISPR THERAPEUTICS AG
- Roman Lvovitch Bogorad
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Formulations for small intestinal delivery of RSV and norovirus ant...
- Patent number 12,186,407
- Issue date Jan 7, 2025
- Vaxart, Inc.
- George Trager
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Systemic gene replacement therapy for treatment of X-linked myotubu...
- Patent number 12,178,856
- Issue date Dec 31, 2024
- Wake Forest University Health Sciences Genethon
- Martin K. Childers
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Methods and compositions relating to the treatment of tumors
- Patent number 12,178,845
- Issue date Dec 31, 2024
- The General Hospital Corporation
- Gary J. Brenner
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Hybrid regulatory elements
- Patent number 12,171,843
- Issue date Dec 24, 2024
- Genethon
- Federico Mingozzi
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Gene therapy for recessive dystrophic epidermolysis bullosa using g...
- Patent number 12,173,314
- Issue date Dec 24, 2024
- The Board of Trustees of the Leland Stanford Junior University
- Zurab Siprashvili
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
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
Optimized expression cassettes for gene therapy
- Patent number 12,151,001
- Issue date Nov 26, 2024
- Tenaya Therapeutics, Inc.
- Huanyu Zhou
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Chimeric protein and related genic transfer technology
- Patent number 12,138,319
- Issue date Nov 12, 2024
- Lucio Pastore
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Methods for treating or alleviating a bone-loss related disease or...
- Patent number 12,133,896
- Issue date Nov 5, 2024
- Kaohsiung Medical University
- Chau-Zen Wang
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Gene therapy delivery of Parkin mutants having increased activity t...
- Patent number 12,133,897
- Issue date Nov 5, 2024
- NysnoBio GT Neurology, LLC
- Jennifer Johnston
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Compositions for treating pathological calcification conditions, an...
- Patent number 12,128,087
- Issue date Oct 29, 2024
- Yale University
- Demetrios Braddock
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Recombinant adeno associated virus encoding clarin-1 and uses thereof
- Patent number 12,129,287
- Issue date Oct 29, 2024
- President and Fellows of Harvard College
- David P. Corey
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Rescuing voltage-gated sodium channel function in inhibitory neurons
- Patent number 12,121,563
- Issue date Oct 22, 2024
- Allen Institute
- John K. Mich
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Methods and materials for GALGT2 gene therapy
- Patent number 12,121,595
- Issue date Oct 22, 2024
- Research Institute at Nationwide Children's Hospital
- Paul Taylor Martin
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Non-invasive optogenetic stimulation method to regulate glucose met...
- Patent number 12,121,743
- Issue date Oct 22, 2024
- Albert Einstein College of Medicine
- Young-Hwan Jo
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Capsid variants and methods of using the same
- Patent number 12,116,385
- Issue date Oct 15, 2024
- DYNO THERAPEUTICS, INC.
- Ina Chen
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Gene therapy for recessive dystrophic epidermolysis bullosa using g...
- Patent number 12,110,504
- Issue date Oct 8, 2024
- The Board of Trustees of the Leland Stanford Junior University
- Zurab Siprashvili
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Models of tauopathy
- Patent number 12,110,502
- Issue date Oct 8, 2024
- REGENERON PHARMACEUTICALS, INC.
- Marine Prissette
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Adeno-associated virus vectors for treatment of Rett syndrome
- Patent number 12,104,163
- Issue date Oct 1, 2024
- Sarepta Therapeutics, Inc.
- Colin O'Banion
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Modulation of neural pathways
- Patent number 12,097,268
- Issue date Sep 24, 2024
- EOS NEUROSCIENCE, INC.
- Alan Horsager
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Gene therapy strategy to restore cardiac electrical and structural...
- Patent number 12,098,385
- Issue date Sep 24, 2024
- The Regents of the University of California
- Farah Sheikh
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Engineered DNA binding proteins
- Patent number 12,083,188
- Issue date Sep 10, 2024
- Encoded Therapeutics, Inc.
- Stephanie Tagliatela
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Vectors for the treatment of Friedreich's ataxia
- Patent number 12,084,673
- Issue date Sep 10, 2024
- FUNDACIÓ INSTITUT D'INVESTIGACIÓ EN CIENCIES DE LA SALUT GERMANS TRIAS I PUJOL
- Antoni Matilla Dueñas
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Lipin-1 as a novel therapy target of muscular dystrophy
- Patent number 12,076,419
- Issue date Sep 3, 2024
- Hongmei Ren
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Disulfide bond stabilized polypeptide compositions and methods of use
- Patent number 12,064,485
- Issue date Aug 20, 2024
- Amicus Therapeutics, Inc.
- Hung Do
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Nucleic acid-polypeptide compositions and methods of inducing exon...
- Patent number 12,064,483
- Issue date Aug 20, 2024
- AVIDITY BIOSCIENCES, INC.
- Arthur A. Levin
- 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
Materials and methods for treatment of Duchenne Muscular Dystrophy
- Patent number 12,053,531
- Issue date Aug 6, 2024
- Vertex Pharmaceuticals Incorporated
- Ami Meda Kabadi
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...

Patents Applicationslast 30 patents

Information Patent Application
PRODUCTION OF RECOMBINANT AAV VECTORS FOR TREATING MUSCULAR DYSTROPHY
- Publication number 20250018059
- Publication date Jan 16, 2025
- Sarepta Therapeutics, Inc.
- Maroof ALAM
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
METHOD FOR DECREASING DEGENERATION OF RETINAL GANGLION CELLS
- Publication number 20250018058
- Publication date Jan 16, 2025
- Icahn School of Medicine at Mount Sinai
- Bo CHEN
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
TREATMENT OF INTERVERTEBRAL DISC DEGENERATION
- Publication number 20250018011
- Publication date Jan 16, 2025
- Kolon TissueGene, Inc.
- Moon Jong Noh
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
GENE THERAPY IN SUCCINIC SEMIALDEHYDE DEHYDROGENASE DEFICIENCY (SSA...
- Publication number 20250018061
- Publication date Jan 16, 2025
- The Children's Medical Center Corporation
- Alexander Rotenberg
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
ADENOVIRUS DELIVERY SYSTEM FOR CANCER TREATMENT
- Publication number 20250018060
- Publication date Jan 16, 2025
- Christiana Care Gene Editing Institute, Inc.
- Eric B. Kmiec
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
METHODS FOR EVALUATING TREATMENTS FOR BESTROPHINOPATHIES
- Publication number 20250009905
- Publication date Jan 9, 2025
- The Trustees of the University of Pennsylvania
- Karina E. Guziewicz
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
NOCICEPTOR-SPECIFIC GENE REGULATORY ELEMENTS FOR THE TREATMENT OF PAIN
- Publication number 20250009906
- Publication date Jan 9, 2025
- President and Fellows of Harvard College
- William Renthal
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
HUMAN MESOTHELIN CHIMERIC ANTIGEN RECEPTORS AND USES THEREOF
- Publication number 20250011461
- Publication date Jan 9, 2025
- Novartis AG
- Gregory Beatty
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
AAV CAPSID VARIANTS AND USES THEREOF
- Publication number 20250001012
- Publication date Jan 2, 2025
- Mathieu Emmanuel Nonnenmacher
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
METHODS FOR THERAPY AND REHABILITATION OF MOVEMENT DISORDERS
- Publication number 20250002555
- Publication date Jan 2, 2025
- City University of Hong Kong
- Jufang He
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
ENGINEERED ADAR-RECRUITING RNAS AND METHODS OF USE THEREOF
- Publication number 20250001011
- Publication date Jan 2, 2025
- Peking University
- Wensheng WEI
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
AAV2-MEDIATED GENE DELIVERY OF SFASL AS A NEUROPROTECTIVE THERAPY I...
- Publication number 20250001015
- Publication date Jan 2, 2025
- University of Massachusetts
- Ann Marshak-Rothstein
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
RETGC Gene Therapy
- Publication number 20250002936
- Publication date Jan 2, 2025
- MEIRAGTX UK II LIMITED
- Anastasios Georgiadis
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
COMPOSITIONS AND METHODS FOR ADENO-ASSOCIATED (AAV) VIRUS DNASE EXP...
- Publication number 20240425879
- Publication date Dec 26, 2024
- CLS THERAPEUTICS, LLC
- Dmitry Genkin
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
METHODS AND COMPOSITIONS FOR DIRECT LINEAGE REPROGRAMMING OF ASTROC...
- Publication number 20240424140
- Publication date Dec 26, 2024
- The Governing Council of the University of Toronto
- Maryam FAIZ
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
METHODS OF TREATING NEURODEGENERATIVE CONDITIONS AND COMPOSITIONS T...
- Publication number 20240424142
- Publication date Dec 26, 2024
- Yale University
- Jaime GRUTZENDLER
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
ENGINEERED MUSCLE AND CENTRAL NERVOUS SYSTEM COMPOSITIONS
- Publication number 20240424143
- Publication date Dec 26, 2024
- THE BROAD INSTITUTE, INC.
- Pardis SABETI
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
GENE THERAPY FOR SPINAL MUSCULAR ATROPHY
- Publication number 20240424144
- Publication date Dec 26, 2024
- University of Massachusetts
- Jun Xie
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
AAV PARTICLES COMPRISING A LIVER-TROPIC CAPSID PROTEIN AND ALPHA-GA...
- Publication number 20240424141
- Publication date Dec 26, 2024
- CANbridge Pharmaceuticals, Inc.
- Yunxiang Zhu
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
CAPSID VARIANTS AND METHODS OF USING THE SAME
- Publication number 20240425551
- Publication date Dec 26, 2024
- DYNO THERAPEUTICS, INC.
- Ina Chen
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
CAPSID VARIANTS AND METHODS OF USING THE SAME
- Publication number 20240425552
- Publication date Dec 26, 2024
- DYNO THERAPEUTICS, INC.
- Ina Chen
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
AAV-MEDIATED THERAPIES FOR VISION LOSS ASSOCIATED WITH FRIEDREICH’S...
- Publication number 20240415983
- Publication date Dec 19, 2024
- University of Florida Research Foundation, Incorporated
- Sanford L. Boye
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
VIRUS TO EXPRESS GAD65 UNDER E2 REGULATORY ELEMENT
- Publication number 20240417713
- Publication date Dec 19, 2024
- NEW YORK UNIVERSITY
- Dan H. Sanes
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
NUCLEIC ACID CONSTRUCTS, VIRAL VECTORS AND VIRAL PARTICLES
- Publication number 20240417434
- Publication date Dec 19, 2024
- UCB Biopharma SRL
- Natalia RODRIGUEZ ALVAREZ
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
GENE EDITING FOR HEMOPHILIA A WITH IMPROVED FACTOR VIII EXPRESSION
- Publication number 20240415981
- Publication date Dec 19, 2024
- CRISPR Therapeutics AG
- Alan Richard Brooks
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
GENE THERAPY WITH THE GENES HOKD AND LDRB FOR CANCER TREATMENT
- Publication number 20240415982
- Publication date Dec 19, 2024
- UNIVERSIDAD DE GRANADA
- Houria BOULAIZ TASSI
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
Precise Excisions of Portions of Exons for Treatment of Duchenne Mu...
- Publication number 20240415984
- Publication date Dec 19, 2024
- VERTEX PHARMACEUTICALS INCORPORATED
- Tudor Fulga
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
CAPSID VARIANTS AND METHODS OF USING THE SAME
- Publication number 20240417430
- Publication date Dec 19, 2024
- DYNO THERAPEUTICS, INC.
- Ina Chen
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
NME2CAS9 INLAID DOMAIN FUSION PROTEINS
- Publication number 20240408238
- Publication date Dec 12, 2024
- University of Massachusetts
- Erik Joseph SONTHEIMER
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
COMPOSITIONS AND METHODS TO PREVENT, INHIBIT OR TREAT NEURODEGENERA...
- Publication number 20240408239
- Publication date Dec 12, 2024
- THE TRUSTEES OF COLUMBIA UNIVERSITY IN THE CITY OF NEW YORK
- Ai Yamamoto
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE

Nucleic acids adapted for tissue specific expression

Industry

Parent Industries

Current Industry

Patents Grantslast 30 patents

Compositions and processes for targeted delivery, expression and mo...

Materials and methods for treatment of hereditary haemochromatosis

Formulations for small intestinal delivery of RSV and norovirus ant...

Systemic gene replacement therapy for treatment of X-linked myotubu...

Methods and compositions relating to the treatment of tumors

Hybrid regulatory elements

Gene therapy for recessive dystrophic epidermolysis bullosa using g...

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

Optimized expression cassettes for gene therapy

Chimeric protein and related genic transfer technology

Methods for treating or alleviating a bone-loss related disease or...

Gene therapy delivery of Parkin mutants having increased activity t...

Compositions for treating pathological calcification conditions, an...

Recombinant adeno associated virus encoding clarin-1 and uses thereof

Rescuing voltage-gated sodium channel function in inhibitory neurons

Methods and materials for GALGT2 gene therapy

Non-invasive optogenetic stimulation method to regulate glucose met...

Capsid variants and methods of using the same

Gene therapy for recessive dystrophic epidermolysis bullosa using g...

Models of tauopathy

Adeno-associated virus vectors for treatment of Rett syndrome

Modulation of neural pathways

Gene therapy strategy to restore cardiac electrical and structural...

Engineered DNA binding proteins

Vectors for the treatment of Friedreich's ataxia

Lipin-1 as a novel therapy target of muscular dystrophy

Disulfide bond stabilized polypeptide compositions and methods of use

Nucleic acid-polypeptide compositions and methods of inducing exon...

Gene therapy

Materials and methods for treatment of Duchenne Muscular Dystrophy

Patents Applicationslast 30 patents

PRODUCTION OF RECOMBINANT AAV VECTORS FOR TREATING MUSCULAR DYSTROPHY

METHOD FOR DECREASING DEGENERATION OF RETINAL GANGLION CELLS

TREATMENT OF INTERVERTEBRAL DISC DEGENERATION

GENE THERAPY IN SUCCINIC SEMIALDEHYDE DEHYDROGENASE DEFICIENCY (SSA...

ADENOVIRUS DELIVERY SYSTEM FOR CANCER TREATMENT

METHODS FOR EVALUATING TREATMENTS FOR BESTROPHINOPATHIES

NOCICEPTOR-SPECIFIC GENE REGULATORY ELEMENTS FOR THE TREATMENT OF PAIN

HUMAN MESOTHELIN CHIMERIC ANTIGEN RECEPTORS AND USES THEREOF

AAV CAPSID VARIANTS AND USES THEREOF

METHODS FOR THERAPY AND REHABILITATION OF MOVEMENT DISORDERS

ENGINEERED ADAR-RECRUITING RNAS AND METHODS OF USE THEREOF

AAV2-MEDIATED GENE DELIVERY OF SFASL AS A NEUROPROTECTIVE THERAPY I...

RETGC Gene Therapy

COMPOSITIONS AND METHODS FOR ADENO-ASSOCIATED (AAV) VIRUS DNASE EXP...

METHODS AND COMPOSITIONS FOR DIRECT LINEAGE REPROGRAMMING OF ASTROC...

METHODS OF TREATING NEURODEGENERATIVE CONDITIONS AND COMPOSITIONS T...

ENGINEERED MUSCLE AND CENTRAL NERVOUS SYSTEM COMPOSITIONS

GENE THERAPY FOR SPINAL MUSCULAR ATROPHY

AAV PARTICLES COMPRISING A LIVER-TROPIC CAPSID PROTEIN AND ALPHA-GA...

CAPSID VARIANTS AND METHODS OF USING THE SAME

CAPSID VARIANTS AND METHODS OF USING THE SAME

AAV-MEDIATED THERAPIES FOR VISION LOSS ASSOCIATED WITH FRIEDREICH’S...

VIRUS TO EXPRESS GAD65 UNDER E2 REGULATORY ELEMENT

NUCLEIC ACID CONSTRUCTS, VIRAL VECTORS AND VIRAL PARTICLES

GENE EDITING FOR HEMOPHILIA A WITH IMPROVED FACTOR VIII EXPRESSION

GENE THERAPY WITH THE GENES HOKD AND LDRB FOR CANCER TREATMENT

Precise Excisions of Portions of Exons for Treatment of Duchenne Mu...

CAPSID VARIANTS AND METHODS OF USING THE SAME

NME2CAS9 INLAID DOMAIN FUSION PROTEINS

COMPOSITIONS AND METHODS TO PREVENT, INHIBIT OR TREAT NEURODEGENERA...