Demonstrated in vivo effect

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C12N2750/14171

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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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C12N2750/14171Demonstrated in vivo effect

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Patents Grantslast 30 patents

Information Patent Grant
Gene therapeutics for treating bone disorders
- Patent number 12,194,108
- Issue date Jan 14, 2025
- UNIVERSITY OF MASSACHUSETTS
- Jae-Hyuck Shim
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Codon optimized otoferlin AAV dual vector gene therapy
- Patent number 12,188,041
- Issue date Jan 7, 2025
- University of Florida Research Foundation, Incorporated
- Frank M. Dyka
- 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
Compositions and methods for impeding transcription of expanded mic...
- Patent number 12,180,499
- Issue date Dec 31, 2024
- University of Florida Research Foundation, Inc.
- Eric Tzy-Shi Wang
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Human PAH expression cassette for treatment of PKU by liver-directe...
- Patent number 12,173,308
- Issue date Dec 24, 2024
- Genzyme Corporation
- Sirkka R. M. Kyostio-Moore
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Cardiac cell reprogramming with myocardin and ASCL1
- Patent number 12,168,778
- Issue date Dec 17, 2024
- Tenaya Therapeutics, Inc.
- Huanyu Zhou
- 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
Expression vector for cholesterol 24-hydrolase in therapy of polygl...
- Patent number 12,138,298
- Issue date Nov 12, 2024
- BRAINVECTIS
- Sandro Alves
- 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
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
Methods to treat mucopolysaccharidosis type II or deficiency in idu...
- Patent number 12,121,567
- Issue date Oct 22, 2024
- Regents of The University of Minnesota
- R. Scott McIvor
- 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
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
Adeno-associated virus with engineered capsid
- Patent number 12,104,165
- Issue date Oct 1, 2024
- Tenaya Therapeutics, Inc.
- Christopher A. Reid
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
High efficiency library-identified AAV vectors
- Patent number 12,091,659
- Issue date Sep 17, 2024
- University of Massachusetts
- Miguel Sena Esteves
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Viral vector constructs for delivery of nucleic acids encoding cyto...
- Patent number 12,071,633
- Issue date Aug 27, 2024
- Kriya Therapeutics, Inc.
- Brian Furmanski
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Adeno-associated virus virion for treatment of Tay-Sachs disease an...
- Patent number 12,064,460
- Issue date Aug 20, 2024
- TOKUSHIMA UNIVERSITY
- Kohji Itoh
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Adeno-associated virus compositions for PAH gene transfer and metho...
- Patent number 12,064,486
- Issue date Aug 20, 2024
- HOMOLOGY MEDICINES, INC.
- Albert Barnes Seymour
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Modified AAV constructs and uses thereof
- Patent number 12,054,715
- Issue date Aug 6, 2024
- University of Massachusetts
- Guangping Gao
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
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
AAV vectors
- Patent number 12,043,848
- Issue date Jul 23, 2024
- Vigeneron GmbH
- Stylianos Michalakis
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Hybrid recombinant adeno-associated virus serotype between AAV9 and...
- Patent number 12,037,362
- Issue date Jul 16, 2024
- Genethon
- Isabelle Richard
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Modified AAV capsids and uses thereof
- Patent number 12,030,914
- Issue date Jul 9, 2024
- Adverum Biotechnologies, Inc.
- Annahita Keravala
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Efficient systemic treatment of dystrophic muscle pathologies
- Patent number 12,023,366
- Issue date Jul 2, 2024
- Genethon
- George Dickson
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Compositions and methods for regulating production of a fusion prot...
- Patent number 12,018,274
- Issue date Jun 25, 2024
- Wyvern Pharmaceuticals Inc.
- Bradley G. Thompson
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Use of Mullerian inhibiting substance (MIS) proteins for contracept...
- Patent number 11,998,590
- Issue date Jun 4, 2024
- The General Hospital Corporation
- Patricia K. Donahoe
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Treatment of mucopolysaccharidosis II with recombinant human iduron...
- Patent number 11,986,515
- Issue date May 21, 2024
- REGENXBIO Inc.
- Stephen Joseph Pakola
- G01 - MEASURING TESTING
Information Patent Grant
Methods and compositions for delivery of immunotherapy agents acros...
- Patent number 11,981,705
- Issue date May 14, 2024
- The Brigham & Women's Hospital, Inc.
- Fengfeng Bei
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Gene therapy for neurodegenerative disorders
- Patent number 11,975,043
- Issue date May 7, 2024
- Genzyme Corporation
- Marco A. Passini
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Antibody-evading virus vectors
- Patent number 11,976,096
- Issue date May 7, 2024
- Ginkgo BioWorks, Inc.
- Daniel McCoy
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...

Patents Applicationslast 30 patents

Information Patent Application
ENGINEERED MUSCLE TARGETING COMPOSITIONS
- Publication number 20250043309
- Publication date Feb 6, 2025
- THE BROAD INSTITUTE, INC.
- Pardis SABETI
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
GENE THERAPY FOR HEARING LOSS
- Publication number 20250041451
- Publication date Feb 6, 2025
- Perceive Biotherapeutics, Inc.
- Derek Welsbie
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
COMPOSITIONS AND METHODS FOR THE TREATMENT OF TAU-RELATED DISORDERS
- Publication number 20250034559
- Publication date Jan 30, 2025
- VOYAGER THERAPEUTICS, INC.
- Mathieu Emmanuel NONNENMACHER
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
HIGH EFFICIENCY LIBRARY-IDENTIFIED AAV VECTORS
- Publication number 20250034555
- Publication date Jan 30, 2025
- University of Massachusetts
- Miguel Sena Esteves
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
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
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
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
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-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
MODIFIED AAV CONSTRUCTS AND USES THEREOF
- Publication number 20240409940
- Publication date Dec 12, 2024
- University of Massachusetts
- Guangping Gao
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
ENGINEERED CIRCULAR RNA CIRCMIR-29B AND USE THEREOF IN PREPARATION...
- Publication number 20240401049
- Publication date Dec 5, 2024
- SHANGHAI UNIVERSITY
- Junjie XIAO
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
RECOMBINANT ADENO-ASSOCIATED VIRUS WITH MODIFIED AAV CAPSID POLYPEP...
- Publication number 20240398992
- Publication date Dec 5, 2024
- Skyline Therapeutics Limited
- Yanqun SHU
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
COMPOSITIONS, KITS AND METHODS FOR TREATMENT OF COMPLEMENT-RELATED...
- Publication number 20240398898
- Publication date Dec 5, 2024
- Trustees of Tufts College
- Rajendra Kumar-Singh
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
ADENO-ASSOCIATED VIRUS VECTORS FOR TREATMENT OF RETT SYNDROME
- Publication number 20240392316
- Publication date Nov 28, 2024
- Sarepta Therapeutics, Inc.
- Colin O'Banion
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
ADENO-ASSOCIATED VECTORS AND VIRIONS TO TREAT GALACTOSEMIA AND METH...
- Publication number 20240390516
- Publication date Nov 28, 2024
- Jaguar Gene Therapy, LLC
- Michael Hughes
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
SYSTEMS AND USES THEREOF FOR THE TREATMENT OF DMD-ASSOCIATED DISEASES
- Publication number 20240384249
- Publication date Nov 21, 2024
- MAMMOTH BIOSCIENCES, INC.
- Lucas Benjamin HARRINGTON
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
SYSTEMIC DELIVERY OF ADENO-ASSOCIATED VIRUS VECTOR EXPRESSING GAMMA...
- Publication number 20240374684
- Publication date Nov 14, 2024
- Sarepta Therapeutics, Inc.
- Louise RODINO-KLAPAC
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
COMPOSITIONS AND METHODS FOR IMPROVED TREATMENT OF X-LINKED MYOTUBU...
- Publication number 20240366785
- Publication date Nov 7, 2024
- Audentes Therapeutics, Inc.
- Weston MILLER
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
Gene Therapy For Neurodegenerative Disorders
- Publication number 20240350584
- Publication date Oct 24, 2024
- Genzyme Corporation
- Marco A. PASSINI
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
GENE THERAPY FOR TUBEROUS SCLEROSIS
- Publication number 20240343768
- Publication date Oct 17, 2024
- The General Hospital Corporation
- Xandra BREAKEFIELD
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
VECTOR ENCODING ROD-DERIVED CONE VIABILITY FACTOR AND HUMAN IGK SIG...
- Publication number 20240342313
- Publication date Oct 17, 2024
- PHARMA CINQ, LLC
- Tianci LUO
- 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
Hybrid Recombinant Adeno-Associated Virus Serotype Between AAV9 and...
- Publication number 20240317814
- Publication date Sep 26, 2024
- Genethon
- Isabelle Richard
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
AAV Vectors
- Publication number 20240318149
- Publication date Sep 26, 2024
- VIGENERON GMBH
- Stylianos MICHALAKIS
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
METHODS AND COMPOSITIONS FOR ADMINISTERING OTOFERLIN DUAL VECTOR SY...
- Publication number 20240309399
- Publication date Sep 19, 2024
- Decibel Therapeutics, Inc.
- Qi-Ying HU
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
MODIFIED AAV CAPSIDS AND USES THEREOF
- Publication number 20240301008
- Publication date Sep 12, 2024
- Adverum Biotechnologies, Inc.
- Annahita Keravala
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
ADENO-ASSOCIATED VIRAL (AAV) VECTORS FOR TISSUE-TARGETED EXPRESSION...
- Publication number 20240285804
- Publication date Aug 29, 2024
- The Brigham & Women's Hospital, Inc.
- Bei Fengfeng
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
Therapeutic Editing
- Publication number 20240279650
- Publication date Aug 22, 2024
- Shape Therapeutics Inc.
- David HUSS
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
USE OF MULLERIAN INHIBITING SUBSTANCE (MIS) PROTEINS FOR CONTRACEPT...
- Publication number 20240277812
- Publication date Aug 22, 2024
- The General Hospital Corporation
- Patricia K. DONAHOE
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
Retinal Disorders
- Publication number 20240279680
- Publication date Aug 22, 2024
- Ikarovec Limited
- Peter Widdowson
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...

Demonstrated in vivo effect

Industry

Parent Industries

Current Industry

Patents Grantslast 30 patents

Gene therapeutics for treating bone disorders

Codon optimized otoferlin AAV dual vector gene therapy

Methods and compositions relating to the treatment of tumors

Compositions and methods for impeding transcription of expanded mic...

Human PAH expression cassette for treatment of PKU by liver-directe...

Cardiac cell reprogramming with myocardin and ASCL1

Optimized expression cassettes for gene therapy

Expression vector for cholesterol 24-hydrolase in therapy of polygl...

Methods of predicting ancestral virus sequences and uses thereof

Recombinant adeno associated virus encoding clarin-1 and uses thereof

Methods to treat mucopolysaccharidosis type II or deficiency in idu...

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

Adeno-associated virus vectors for treatment of Rett syndrome

Adeno-associated virus with engineered capsid

High efficiency library-identified AAV vectors

Viral vector constructs for delivery of nucleic acids encoding cyto...

Adeno-associated virus virion for treatment of Tay-Sachs disease an...

Adeno-associated virus compositions for PAH gene transfer and metho...

Modified AAV constructs and uses thereof

Methods and compositions for dual glycan binding AAV vectors

AAV vectors

Hybrid recombinant adeno-associated virus serotype between AAV9 and...

Modified AAV capsids and uses thereof

Efficient systemic treatment of dystrophic muscle pathologies

Compositions and methods for regulating production of a fusion prot...

Use of Mullerian inhibiting substance (MIS) proteins for contracept...

Treatment of mucopolysaccharidosis II with recombinant human iduron...

Methods and compositions for delivery of immunotherapy agents acros...

Gene therapy for neurodegenerative disorders

Antibody-evading virus vectors

Patents Applicationslast 30 patents

ENGINEERED MUSCLE TARGETING COMPOSITIONS

GENE THERAPY FOR HEARING LOSS

COMPOSITIONS AND METHODS FOR THE TREATMENT OF TAU-RELATED DISORDERS

HIGH EFFICIENCY LIBRARY-IDENTIFIED AAV VECTORS

PRODUCTION OF RECOMBINANT AAV VECTORS FOR TREATING MUSCULAR DYSTROPHY

METHODS FOR EVALUATING TREATMENTS FOR BESTROPHINOPATHIES

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

GENE THERAPY FOR SPINAL MUSCULAR ATROPHY

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

MODIFIED AAV CONSTRUCTS AND USES THEREOF

ENGINEERED CIRCULAR RNA CIRCMIR-29B AND USE THEREOF IN PREPARATION...

RECOMBINANT ADENO-ASSOCIATED VIRUS WITH MODIFIED AAV CAPSID POLYPEP...

COMPOSITIONS, KITS AND METHODS FOR TREATMENT OF COMPLEMENT-RELATED...

ADENO-ASSOCIATED VIRUS VECTORS FOR TREATMENT OF RETT SYNDROME

ADENO-ASSOCIATED VECTORS AND VIRIONS TO TREAT GALACTOSEMIA AND METH...

SYSTEMS AND USES THEREOF FOR THE TREATMENT OF DMD-ASSOCIATED DISEASES

SYSTEMIC DELIVERY OF ADENO-ASSOCIATED VIRUS VECTOR EXPRESSING GAMMA...

COMPOSITIONS AND METHODS FOR IMPROVED TREATMENT OF X-LINKED MYOTUBU...

Gene Therapy For Neurodegenerative Disorders

GENE THERAPY FOR TUBEROUS SCLEROSIS

VECTOR ENCODING ROD-DERIVED CONE VIABILITY FACTOR AND HUMAN IGK SIG...

METHODS AND COMPOSITIONS FOR DUAL GLYCAN BINDING AAV VECTORS

Hybrid Recombinant Adeno-Associated Virus Serotype Between AAV9 and...

AAV Vectors

METHODS AND COMPOSITIONS FOR ADMINISTERING OTOFERLIN DUAL VECTOR SY...

MODIFIED AAV CAPSIDS AND USES THEREOF

ADENO-ASSOCIATED VIRAL (AAV) VECTORS FOR TISSUE-TARGETED EXPRESSION...

Therapeutic Editing

USE OF MULLERIAN INHIBITING SUBSTANCE (MIS) PROTEINS FOR CONTRACEPT...

Retinal Disorders