cell type or tissue specific enhancer/promoter combination

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C12N2830/008

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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 C12N2830/00Vector systems having a special element relevant for transcription

Current Industry

C12N2830/008cell type or tissue specific enhancer/promoter combination

Industries

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Patents Grantslast 30 patents

Information Patent Grant
Factor IX encoding nucleotides
- Patent number 12,209,262
- Issue date Jan 28, 2025
- UCL BUSINESS LTD
- Amit Nathwani
- 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
Method for delivering RNA to neurons to treat herpes infections
- Patent number 12,156,919
- Issue date Dec 3, 2024
- University of Florida Research Foundation, Incorporated
- David C. Bloom
- 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
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
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
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
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
Methods of treating solid or lymphatic tumors by combination therapy
- Patent number 12,090,183
- Issue date Sep 17, 2024
- CG ONCOLOGY, INC.
- Alex Wah Hin Yeung
- C07 - ORGANIC CHEMISTRY
Information Patent Grant
Non-human animals models of retinoschisis
- Patent number 12,082,565
- Issue date Sep 10, 2024
- REGENERON PHARMACEUTICALS, INC.
- Susannah Brydges
- A01 - AGRICULTURE FORESTRY ANIMAL HUSBANDRY HUNTING TRAPPING FISHING
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
Methods for using transcription-dependent directed evolution of AAV...
- Patent number 12,084,654
- Issue date Sep 10, 2024
- Oregon Health & Science University
- Hiroyuki Nakai
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Methods for using transcription-dependent directed evolution of AAV...
- Patent number 12,084,653
- Issue date Sep 10, 2024
- Oregon Health & Science University
- Hiroyuki Nakai
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
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
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
Cell therapy with lentiviral transduced CSF2RA transgene in the tre...
- Patent number 12,043,844
- Issue date Jul 23, 2024
- CHILDREN'S HOSPITAL MEDICAL CENTER
- Bruce C. Trapnell
- A01 - AGRICULTURE FORESTRY ANIMAL HUSBANDRY HUNTING TRAPPING FISHING
Information Patent Grant
Composition for treatment of Crigler-Najjar syndrome
- Patent number 12,023,386
- Issue date Jul 2, 2024
- The Trustees of the University of Pennsylvania
- Anna P. Tretiakova
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Compositions and methods for treating non-age-associated hearing im...
- Patent number 11,993,777
- Issue date May 28, 2024
- Akouos, Inc.
- Emmanuel John Simons
- C07 - ORGANIC CHEMISTRY
Information Patent Grant
Adeno associated virus vectors for the treatment of hunter disease
- Patent number 11,981,912
- Issue date May 14, 2024
- Takeda Pharma ceutical Company Limited
- Vivian Choi
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Muscle-specific nucleic acid regulatory elements and methods and us...
- Patent number 11,975,078
- Issue date May 7, 2024
- Universiteit Gent
- Thierry Vandendriessche
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Optimized RPE65 promoter and coding sequences
- Patent number 11,969,478
- Issue date Apr 30, 2024
- UCL BUSINESS LTD
- Alexander Smith
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Micro-dystrophins and related methods of use
- Patent number 11,958,888
- Issue date Apr 16, 2024
- University of Washington
- Jeffrey S. Chamberlain
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Methods of treating phenylketonuria
- Patent number 11,952,585
- Issue date Apr 9, 2024
- HOMOLOGY MEDICINES, INC.
- Omar L. Francone
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Therapeutical tools and methods for treating blindness by targeting...
- Patent number 11,931,427
- Issue date Mar 19, 2024
- FRIEDRICH MIESCHER INSTITUTE FOR BIOMEDICAL RESEARCH
- Josephine Juettner
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Engineered muscle targeting compositions
- Patent number 11,920,150
- Issue date Mar 5, 2024
- The Broad Institute, Inc.
- Pardis Sabeti
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Diaphragm-specific nucleic acid regulatory elements and methods and...
- Patent number 11,920,149
- Issue date Mar 5, 2024
- Vrije Universiteit Brussel
- Lay Khim Chuah
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Embryonic cell cultures and methods of using the same
- Patent number 11,913,015
- Issue date Feb 27, 2024
- University of Maryland, College Park
- Chi-Hun Park
- A01 - AGRICULTURE FORESTRY ANIMAL HUSBANDRY HUNTING TRAPPING FISHING
Information Patent Grant
Treatment of diseases by liver expression of an enzyme which has a...
- Patent number 11,905,522
- Issue date Feb 20, 2024
- CLS THERAPEUTICS LIMITED
- Dmitry Dmitrievich Genkin
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Restoration of visual responses by in vivo delivery of rhodopsin nu...
- Patent number 11,883,463
- Issue date Jan 30, 2024
- Wayne State University
- Zhuo-Hua Pan
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...

Patents Applicationslast 30 patents

Information Patent Application
ENHANCERS FOR DIRECTED EXPRESSION OF GENES IN NEURONAL CELL POPULAT...
- Publication number 20250032646
- Publication date Jan 30, 2025
- THE BROAD INSTITUTE, INC.
- Jordane Dimidschstein
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
SYNTHETIC RECEPTORS
- Publication number 20250032643
- Publication date Jan 30, 2025
- UCL Business LTD
- Dimitri KULLMANN
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
Compositions and Methods for Cell-Specific Expression of Target Genes
- Publication number 20250025575
- Publication date Jan 23, 2025
- The Trustees of Princeton University
- Carola Maturana
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
METHODS AND MATERIALS FOR GALGT2 GENE THERAPY
- Publication number 20250025578
- Publication date Jan 23, 2025
- Research Institute at Nationwide Children's Hospital
- Paul Taylor Martin
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
COMPOSITIONS AND METHODS FOR TREATING A MUSCULAR DYSTROPHY
- Publication number 20250019721
- Publication date Jan 16, 2025
- ULTRAGENYX PHARMACEUTICAL INC.
- Matthew Scott Fuller
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
COMPOSITIONS COMPRISING KOZAK SEQUENCES SELECTED FOR ENHANCED EXPRE...
- Publication number 20250019722
- Publication date Jan 16, 2025
- AavantiBio, Inc.
- Widler Casy
- 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
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
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
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
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
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
AUF1 COMBINATION THERAPIES FOR TREATMENT OF MUSCLE DEGENERATIVE DIS...
- Publication number 20240409955
- Publication date Dec 12, 2024
- NEW YORK UNIVERSITY
- Dounia ABBADI
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
ENGINEERED DNA BINDING PROTEINS
- Publication number 20240408242
- Publication date Dec 12, 2024
- Encoded Therapeutics, Inc.
- Stephanie TAGLIATELA
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
PRODUCTS AND METHODS FOR MYELIN PROTEIN ZERO SILENCING AND TREATING...
- Publication number 20240401046
- Publication date Dec 5, 2024
- Research Institute at Nationwide Children's Hospital
- Scott Quenton Harper
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
LIVER-SPECIFIC EXPRESSION CASSETTES, VECTORS AND USES THEREOF FOR E...
- Publication number 20240398990
- Publication date Dec 5, 2024
- GENERATION BIO CO.
- Jessica Lynn Keenan
- 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
GENE THERAPY FOR DUCHENNE MUSCULAR DYSTROPHY
- Publication number 20240390522
- Publication date Nov 28, 2024
- University of Florida Research Foundation, Incorporated
- Hugh Lee Sweeney
- 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
EMBRYONIC CELL CULTURES AND METHODS OF USING THE SAME
- Publication number 20240384302
- Publication date Nov 21, 2024
- University of Maryland, College Park
- Chi-Hun Park
- A01 - AGRICULTURE FORESTRY ANIMAL HUSBANDRY HUNTING TRAPPING FISHING
Information Patent Application
TARGETED GENE THERAPY FOR DM-1 MYOTONIC DYSTROPHY
- Publication number 20240384271
- Publication date Nov 21, 2024
- Genzyme Corporation
- Seng Cheng
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
SYSTEMS AND METHODS FOR THE TARGETED PRODUCTION OF A THERAPEUTIC PR...
- Publication number 20240382623
- Publication date Nov 21, 2024
- Oisin Biotechnologies, Inc.
- Matthew Rein Scholz
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
FRATAXIN GENE THERAPY
- Publication number 20240376497
- Publication date Nov 14, 2024
- Astellas Gene Therapies, Inc.
- Dwaipayan SEN
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
AN ENGINEERED YEAST CELL FOR THE DELIVERY OF ANTIBIOTIC-INACTIVATIN...
- Publication number 20240368575
- Publication date Nov 7, 2024
- JEAN-DENIS DOCQUIER
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
Compositions For and Methods of Editing the Genome
- Publication number 20240368587
- Publication date Nov 7, 2024
- DUKE UNIVERSITY
- Dwight Koeberl
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
HYBRID PROMOTERS FOR GENE EXPRESSION IN MUSCLES AND IN THE CNS
- Publication number 20240350670
- Publication date Oct 24, 2024
- GENETHON
- GIUSEPPE RONZITTI
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
ENGINEERED ITR SEQUENCES AND METHODS OF USE
- Publication number 20240350671
- Publication date Oct 24, 2024
- Bioverativ Therapeutics Inc.
- Ajay Maghodia
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
TROPONIN C (TNNC1) GENE THERAPY USING AAV VECTOR
- Publication number 20240335566
- Publication date Oct 10, 2024
- Spacecraft Seven, LLC
- Christopher Dean HERZOG
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
DIAPHRAGM-SPECIFIC NUCLEIC ACID REGULATORY ELEMENTS AND METHODS AND...
- Publication number 20240327864
- Publication date Oct 3, 2024
- VRIJE UNIVESITEIT BRUSSEL
- Lay Khim CHUAH
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
ANELLOVIRUS CONSTRUCTS COMPRISING RECOMBINATION SITES AND CROSS-PAC...
- Publication number 20240327867
- Publication date Oct 3, 2024
- FLAGSHIP PIONEERING INNOVATIONS V, INC.
- Joseph Michael Cabral
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...

cell type or tissue specific enhancer/promoter combination

Industry

Parent Industries

Current Industry

Patents Grantslast 30 patents

Factor IX encoding nucleotides

Hybrid regulatory elements

Method for delivering RNA to neurons to treat herpes infections

Optimized expression cassettes for gene therapy

CRISPR therapy

Chimeric protein and related genic transfer technology

Rescuing voltage-gated sodium channel function in inhibitory neurons

Methods and materials for GALGT2 gene therapy

Adeno-associated virus vectors for treatment of Rett syndrome

Methods of treating solid or lymphatic tumors by combination therapy

Non-human animals models of retinoschisis

Engineered DNA binding proteins

Methods for using transcription-dependent directed evolution of AAV...

Methods for using transcription-dependent directed evolution of AAV...

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

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

Cell therapy with lentiviral transduced CSF2RA transgene in the tre...

Composition for treatment of Crigler-Najjar syndrome

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

Adeno associated virus vectors for the treatment of hunter disease

Muscle-specific nucleic acid regulatory elements and methods and us...

Optimized RPE65 promoter and coding sequences

Micro-dystrophins and related methods of use

Methods of treating phenylketonuria

Therapeutical tools and methods for treating blindness by targeting...

Engineered muscle targeting compositions

Diaphragm-specific nucleic acid regulatory elements and methods and...

Embryonic cell cultures and methods of using the same

Treatment of diseases by liver expression of an enzyme which has a...

Restoration of visual responses by in vivo delivery of rhodopsin nu...

Patents Applicationslast 30 patents

ENHANCERS FOR DIRECTED EXPRESSION OF GENES IN NEURONAL CELL POPULAT...

SYNTHETIC RECEPTORS

Compositions and Methods for Cell-Specific Expression of Target Genes

METHODS AND MATERIALS FOR GALGT2 GENE THERAPY

COMPOSITIONS AND METHODS FOR TREATING A MUSCULAR DYSTROPHY

COMPOSITIONS COMPRISING KOZAK SEQUENCES SELECTED FOR ENHANCED EXPRE...

NOCICEPTOR-SPECIFIC GENE REGULATORY ELEMENTS FOR THE TREATMENT OF PAIN

RETGC Gene Therapy

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

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

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

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

AUF1 COMBINATION THERAPIES FOR TREATMENT OF MUSCLE DEGENERATIVE DIS...

ENGINEERED DNA BINDING PROTEINS

PRODUCTS AND METHODS FOR MYELIN PROTEIN ZERO SILENCING AND TREATING...

LIVER-SPECIFIC EXPRESSION CASSETTES, VECTORS AND USES THEREOF FOR E...

ADENO-ASSOCIATED VIRUS VECTORS FOR TREATMENT OF RETT SYNDROME

GENE THERAPY FOR DUCHENNE MUSCULAR DYSTROPHY

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

EMBRYONIC CELL CULTURES AND METHODS OF USING THE SAME

TARGETED GENE THERAPY FOR DM-1 MYOTONIC DYSTROPHY

SYSTEMS AND METHODS FOR THE TARGETED PRODUCTION OF A THERAPEUTIC PR...

FRATAXIN GENE THERAPY

AN ENGINEERED YEAST CELL FOR THE DELIVERY OF ANTIBIOTIC-INACTIVATIN...

Compositions For and Methods of Editing the Genome

HYBRID PROMOTERS FOR GENE EXPRESSION IN MUSCLES AND IN THE CNS

ENGINEERED ITR SEQUENCES AND METHODS OF USE

TROPONIN C (TNNC1) GENE THERAPY USING AAV VECTOR

DIAPHRAGM-SPECIFIC NUCLEIC ACID REGULATORY ELEMENTS AND METHODS AND...

ANELLOVIRUS CONSTRUCTS COMPRISING RECOMBINATION SITES AND CROSS-PAC...