characterised by an aspect of the delivery route

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A61K48/0075

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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

Current Industry

A61K48/0075characterised by an aspect of the delivery route

Industries

Patents Grantslast 30 patents

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
Collagen-mimetic peptide mediated delivery of nucleic acid carriers...
- Patent number 12,171,809
- Issue date Dec 24, 2024
- University of Delaware
- Kristi Kiick
- 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 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
Immunostimulating vector system
- Patent number 12,133,898
- Issue date Nov 5, 2024
- Provecs Medical GmbH
- Frank Schnieders
- 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
Lipid nanoparticle compositions and methods for mRNA delivery
- Patent number 12,121,592
- Issue date Oct 22, 2024
- Translate Bio, Inc.
- Frank DeRosa
- 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
Methods and compositions for nucleic acid and protein payload delivery
- Patent number 12,123,012
- Issue date Oct 22, 2024
- LIGANDAL, INC.
- Andre Ronald Watson
- B82 - NANO-TECHNOLOGY
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
Satiation peptide administration
- Patent number 12,109,251
- Issue date Oct 8, 2024
- University of Florida Research Foundation, Incorporated
- Sergei Zolotukhin
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Methods for expressing proteins in axons
- Patent number 12,103,952
- Issue date Oct 1, 2024
- Cornell University
- Samie R. Jaffrey
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Methods for efficient delivery of therapeutic molecules in vitro an...
- Patent number 12,102,691
- Issue date Oct 1, 2024
- Massachusetts Eye and Ear Infirmary
- Zheng-Yi Chen
- C07 - ORGANIC CHEMISTRY
Information Patent Grant
Methods and compositions for treatment of ocular disorders and blin...
- Patent number 12,097,267
- Issue date Sep 24, 2024
- The Trustees of the University of Pennsylvania
- Jeannette Bennicelli
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
System and method for controlling oxygen delivery to implanted cells
- Patent number 12,090,300
- Issue date Sep 17, 2024
- Giner, Inc.
- Simon G. Stone
- C25 - ELECTROLYTIC OR ELECTROPHORETIC PROCESSES APPARATUS THEREFOR
Information Patent Grant
Rabies vaccine
- Patent number 12,083,190
- Issue date Sep 10, 2024
- CureVac SE
- Margit Schnee
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
System for gas treatment of a cell implant
- Patent number 12,070,383
- Issue date Aug 27, 2024
- Giner, Inc.
- Linda Tempelman
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Compositions and methods for treating non-age-associated hearing im...
- Patent number 12,071,627
- Issue date Aug 27, 2024
- Akouos, Inc.
- Emmanuel John Simons
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Injection of single-stranded or self-complementary adeno-associated...
- Patent number 12,070,510
- Issue date Aug 27, 2024
- Emory University
- Anthony Donsante
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Systems and methods for left ventricular unloading in biologic ther...
- Patent number 12,064,120
- Issue date Aug 20, 2024
- Abiomed, Inc.
- Roger J Hajjar
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
System and method of performing treatment along a lumen network
- Patent number 12,059,214
- Issue date Aug 13, 2024
- Covidien LP
- William S. Krimsky
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Delivery of NRF2 as therapy for protection against reactive oxygen...
- Patent number 12,060,395
- Issue date Aug 13, 2024
- University of Florida Research Foundation, Incorporated
- Cristhian J. Ildefonso
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Gene therapy for neurodegenerative disorders
- Patent number 12,049,626
- Issue date Jul 30, 2024
- Prevail Therapeutics, Inc.
- Asa Abeliovich
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Methods and compositions for inhibiting oxidative stress
- Patent number 12,042,546
- Issue date Jul 23, 2024
- President and Fellows of Harvard College
- Constance L. Cepko
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
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
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
Gene editing for hemophilia A with improved factor VIII expression
- Patent number 12,016,932
- Issue date Jun 25, 2024
- CRISPR THERAPEUTICS AG
- Alan Richard Brooks
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Riboswitch modulated gene therapy for retinal diseases
- Patent number 12,012,602
- Issue date Jun 18, 2024
- The Medical College of Wisconsin, Inc.
- Daniel M. Lipinski
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
System and method of performing treatment along a lumen network
- Patent number 12,004,819
- Issue date Jun 11, 2024
- Covidien LP
- William S. Krimsky
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE

Patents Applicationslast 30 patents

Information Patent Application
COMPOSITIONS USEFUL IN TREATMENT OF CDKL5 DEFICIENCY DISORDER (CDD)
- Publication number 20240425882
- Publication date Dec 26, 2024
- The Trustees of the University of Pennsylvania
- James M. Wilson
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
Biomarkers For Lysosomal Storage Diseases
- Publication number 20240424137
- Publication date Dec 26, 2024
- Amicus Therapeutics, Inc.
- Jon Brudvig
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
COMPOSITIONS, SYSTEMS, AND METHODS RELATED TO TRANSGENIC HOOKWORMS...
- Publication number 20240424030
- Publication date Dec 26, 2024
- Washington University
- Makedonka Mitreva
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
CRISPR/CAS-RELATED METHODS AND COMPOSITIONS FOR KNOCKING OUT C5
- Publication number 20240415980
- Publication date Dec 19, 2024
- Regeneron Pharmaceuticals, Inc.
- Kishor Devalaraja-Narashimha
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
MESSENGER RNA THERAPY FOR TREATMENT OF ARTICULAR DISEASE
- Publication number 20240415958
- Publication date Dec 19, 2024
- Translate Bio, Inc.
- Frank DeRosa
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
ANELLOVECTORS FOR DELIVERY OF EFFECTORS TO THE CENTRAL NERVOUS SYSTEM
- Publication number 20240415978
- Publication date Dec 19, 2024
- FLAGSHIP PIONEERING INNOVATIONS V, INC.
- Roger Joseph Hajjar
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
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
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
COMPOSITIONS FOR GENOME EDITING AND METHODS OF USE THEREOF
- Publication number 20240415939
- Publication date Dec 19, 2024
- Arizona Board of Regents on behalf of The University of Arizona
- Arun Dhar
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
MODIFIED POLYNUCLEOTIDES FOR THE PRODUCTION OF SECRETED PROTEINS
- Publication number 20240409597
- Publication date Dec 12, 2024
- ModernaTX, Inc.
- Antonin DE FOUGEROLLES
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
METHODS FOR UPREGULATING SHANK3 EXPRESSION
- Publication number 20240408243
- Publication date Dec 12, 2024
- Massachusetts Institute of Technology
- Guoping Feng
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
TREATMENT OF HEREDITARY ANGIOEDEMA WITH AAV GENE THERAPY VECTORS AN...
- Publication number 20240408240
- Publication date Dec 12, 2024
- BioMarin Pharmaceutical Inc.
- Peter COLOSI
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
METHOD AND PHARMACEUTICAL COMPOSITION FOR TREATING A CARTILAGE DAMA...
- Publication number 20240408237
- Publication date Dec 12, 2024
- Far Eastern Memorial Hospital
- Hsiu-Jung LIAO
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
POLYNUCLEOTIDE COMPOSITIONS, RELATED FORMULATIONS, AND METHODS OF U...
- Publication number 20240398983
- Publication date Dec 5, 2024
- ReCode Therapeutics, Inc.
- Vladimir Kharitonov
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
TREATMENT OF HSV-2 USING A MEGANUCLEASE
- Publication number 20240398984
- Publication date Dec 5, 2024
- Fred Hutchinson Cancer Center
- Keith R. Jerome
- 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
COMPOSITIONS AND METHODS FOR TREATING NON-AGE-ASSOCIATED HEARING IM...
- Publication number 20240384276
- Publication date Nov 21, 2024
- Akouos, Inc.
- Emmanuel John Simons
- A01 - AGRICULTURE FORESTRY ANIMAL HUSBANDRY HUNTING TRAPPING FISHING
Information Patent Application
GENE THERAPY CONSTRUCT FOR TREATING POMPE DISEASE, PHARMACEUTICAL C...
- Publication number 20240384297
- Publication date Nov 21, 2024
- BEIJING GENECRADLE PHARMACEUTICAL CO., LTD.
- Xiaobing WU
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
METHODS AND MATERIALS FOR NT-3 GENE THERAPY
- Publication number 20240383956
- Publication date Nov 21, 2024
- Research Institute at Nationwide Children's Hospital
- Zarife Sahenk
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
VIRUS COMPOSITIONS TO EFFICIENTLY TARGET THE NERVOUS SYSTEM AND THE...
- Publication number 20240382617
- Publication date Nov 21, 2024
- CAPSIDA BIOTHERAPEUTICS, INC.
- Viviana Gradinaru
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
COMPOSITIONS AND METHODS FOR TREATING LEBER'S HEREDITARY OPTIC NEUR...
- Publication number 20240376164
- Publication date Nov 14, 2024
- Wuhan Neurophth Biotechnology Limited Company
- Bin LI
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
DRIVING AXON REGENERATION BY NRF2 OVEREXPRESSION AND EDARAVONE APPL...
- Publication number 20240374570
- Publication date Nov 14, 2024
- THE HONG KONG UNIVERSITY OF SCIENCE AND TECHNOLOGY
- Kai LIU
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
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
CAPSID VARIANTS AND METHODS OF USING THE SAME
- Publication number 20240368228
- Publication date Nov 7, 2024
- DYNO THERAPEUTICS, INC.
- Sylvain Lapan
- 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
COMPOSITIONS AND SYSTEMS FOR RNA-PROGRAMMABLE CELL EDITING AND METH...
- Publication number 20240366792
- Publication date Nov 7, 2024
- DUKE UNIVERSITY
- Z. Josh Huang
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
VIVO GENE THERAPY USING INTRAOSSEOUS DELIVERY OF A LENTIVIRALGENE C...
- Publication number 20240366797
- Publication date Nov 7, 2024
- SEATTLE CHILDREN'S HOSPITAL D/B/A SEATTLE CHILDREN'S RESEARCH INSTITUTE
- Carol Hsing MIAO
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
ADENO-ASSOCIATED VECTORS AND VIRIONS TO TREAT GALACTOSEMIA AND METH...
- Publication number 20240358856
- Publication date Oct 31, 2024
- Jaguar Gene Therapy, LLC
- Michael Hughes
- 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
METHODS AND COMPOSITIONS FOR INHIBITING OXIDATIVE STRESS
- Publication number 20240350673
- Publication date Oct 24, 2024
- President and Fellows of Harvard College
- Constance L. Cepko
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...

characterised by an aspect of the delivery route

Industry

Parent Industries

Current Industry

Patents Grantslast 30 patents

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

Collagen-mimetic peptide mediated delivery of nucleic acid carriers...

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

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

Immunostimulating vector system

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

Lipid nanoparticle compositions and methods for mRNA delivery

Methods and materials for GALGT2 gene therapy

Methods and compositions for nucleic acid and protein payload delivery

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

Satiation peptide administration

Methods for expressing proteins in axons

Methods for efficient delivery of therapeutic molecules in vitro an...

Methods and compositions for treatment of ocular disorders and blin...

System and method for controlling oxygen delivery to implanted cells

Rabies vaccine

System for gas treatment of a cell implant

Compositions and methods for treating non-age-associated hearing im...

Injection of single-stranded or self-complementary adeno-associated...

Systems and methods for left ventricular unloading in biologic ther...

System and method of performing treatment along a lumen network

Delivery of NRF2 as therapy for protection against reactive oxygen...

Gene therapy for neurodegenerative disorders

Methods and compositions for inhibiting oxidative stress

AAV vectors

Modified AAV capsids and uses thereof

Efficient systemic treatment of dystrophic muscle pathologies

Gene editing for hemophilia A with improved factor VIII expression

Riboswitch modulated gene therapy for retinal diseases

System and method of performing treatment along a lumen network

Patents Applicationslast 30 patents

COMPOSITIONS USEFUL IN TREATMENT OF CDKL5 DEFICIENCY DISORDER (CDD)

Biomarkers For Lysosomal Storage Diseases

COMPOSITIONS, SYSTEMS, AND METHODS RELATED TO TRANSGENIC HOOKWORMS...

CRISPR/CAS-RELATED METHODS AND COMPOSITIONS FOR KNOCKING OUT C5

MESSENGER RNA THERAPY FOR TREATMENT OF ARTICULAR DISEASE

ANELLOVECTORS FOR DELIVERY OF EFFECTORS TO THE CENTRAL NERVOUS SYSTEM

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

GENE EDITING FOR HEMOPHILIA A WITH IMPROVED FACTOR VIII EXPRESSION

COMPOSITIONS FOR GENOME EDITING AND METHODS OF USE THEREOF

MODIFIED POLYNUCLEOTIDES FOR THE PRODUCTION OF SECRETED PROTEINS

METHODS FOR UPREGULATING SHANK3 EXPRESSION

TREATMENT OF HEREDITARY ANGIOEDEMA WITH AAV GENE THERAPY VECTORS AN...

METHOD AND PHARMACEUTICAL COMPOSITION FOR TREATING A CARTILAGE DAMA...

POLYNUCLEOTIDE COMPOSITIONS, RELATED FORMULATIONS, AND METHODS OF U...

TREATMENT OF HSV-2 USING A MEGANUCLEASE

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

COMPOSITIONS AND METHODS FOR TREATING NON-AGE-ASSOCIATED HEARING IM...

GENE THERAPY CONSTRUCT FOR TREATING POMPE DISEASE, PHARMACEUTICAL C...

METHODS AND MATERIALS FOR NT-3 GENE THERAPY

VIRUS COMPOSITIONS TO EFFICIENTLY TARGET THE NERVOUS SYSTEM AND THE...

COMPOSITIONS AND METHODS FOR TREATING LEBER'S HEREDITARY OPTIC NEUR...

DRIVING AXON REGENERATION BY NRF2 OVEREXPRESSION AND EDARAVONE APPL...

AAV CAPSID VARIANTS AND USES THEREOF

CAPSID VARIANTS AND METHODS OF USING THE SAME

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

COMPOSITIONS AND SYSTEMS FOR RNA-PROGRAMMABLE CELL EDITING AND METH...

VIVO GENE THERAPY USING INTRAOSSEOUS DELIVERY OF A LENTIVIRALGENE C...

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

Gene Therapy For Neurodegenerative Disorders

METHODS AND COMPOSITIONS FOR INHIBITING OXIDATIVE STRESS