cell type or tissue specific enhancer/promoter combination

Industry

CPC
C12N2830/008

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Parent Industries

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

Patents Grantslast 30 patents

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...
Information Patent Grant
Expression cassette with a SynP160 promoter
- Patent number 11,883,507
- Issue date Jan 30, 2024
- FRIEDRICH MIESCHER INSTITUTE FOR BIOMEDICAL RESEARCH
- Dominik Hartl
- 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
Targeted gene insertion for improved immune cells therapy
- Patent number 11,873,511
- Issue date Jan 16, 2024
- Cellectis
- Brian Busser
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
SYNP161, a promoter for the expression of genes
- Patent number 11,857,642
- Issue date Jan 2, 2024
- FRIEDRICH MIESCHER INSTITUTE FOR BIOMEDICAL RESEARCH
- Dominik Hartl
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Recombinant adenoviruses and stem cells comprising same
- Patent number 11,850,215
- Issue date Dec 26, 2023
- GENEMEDICINE CO., LTD.
- Chae Ok Yun
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Lineage reporter synthetic chromosomes and methods of use
- Patent number 11,851,719
- Issue date Dec 26, 2023
- CarryGenes Bioengineering, LLC
- Edward Perkins
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Methods of treating muscular dystrophy
- Patent number 11,820,972
- Issue date Nov 21, 2023
- Research Institute at Nationwide Children's Hospital
- Louise Rodino-Klapac
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Selective gene therapy expression system
- Patent number 11,819,478
- Issue date Nov 21, 2023
- Genethon
- Ana Maria Buj Bello
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Porcine Thy1 gene promoter specifically expressed in neurons
- Patent number 11,820,998
- Issue date Nov 21, 2023
- JEJU NATIONAL UNIVERSITY INDUSTRY-ACADEMIC COOPERATION FOUNDATION
- Se Pill Park
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Lentiviral vector for treating hemoglobin disorders
- Patent number 11,813,316
- Issue date Nov 14, 2023
- The United States of America, as represented by the Secretary, Department of...
- Naoya Uchida
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Compositions and methods for treating beta-hemoglobinopathies
- Patent number 11,795,461
- Issue date Oct 24, 2023
- CSL Behring Gene Therapy, Inc.
- Jeffrey Ahlers
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Expression vectors comprising engineered genes
- Patent number 11,779,658
- Issue date Oct 10, 2023
- UCL BUSINESS LTD
- Stephanie Schorge
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Compositions and methods for treating non-age-associated hearing im...
- Patent number 11,781,145
- Issue date Oct 10, 2023
- Akouos, Inc.
- Emmanuel John Simons
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Gene-therapy vectors for treating cardiomyopathy
- Patent number 11,773,408
- Issue date Oct 3, 2023
- Lucie Carrier
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Widespread gene delivery to motor neurons using peripheral injectio...
- Patent number 11,767,538
- Issue date Sep 26, 2023
- Genethon
- Martine Barkats
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Globin gene therapy for treating hemoglobinopathies
- Patent number 11,753,654
- Issue date Sep 12, 2023
- Memorial Sloan Kettering Cancer Center
- Michel Sadelain
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
High-transducing HSV vectors
- Patent number 11,753,653
- Issue date Sep 12, 2023
- Periphagen, Inc.
- David M. Krisky
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Primate retinal pigment epithelium cell-specific promoter
- Patent number 11,739,349
- Issue date Aug 29, 2023
- FRIEDRICH MIESCHER INSTITUTE FOR BIOMEDICAL RESEARCH
- Josephine Juettner
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Gene expression system
- Patent number 11,737,436
- Issue date Aug 29, 2023
- Oxitec Limited
- Martha Koukidou
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...

Patents Applicationslast 30 patents

Information Patent Application
PROMOTER FOR SPECIFIC EXPRESSION OF GENES IN ROD PHOTORECEPTORS
- Publication number 20240150791
- Publication date May 9, 2024
- Institute of Molecular and Clinical Ophthalmology Basel (IOB)
- Botond ROSKA
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
LIVER SPECIFIC PRODUCTION OF ENPP1 OR ENPP3
- Publication number 20240148907
- Publication date May 9, 2024
- Inozyme Pharma, Inc.
- Steven Jungles
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
CONFERRING DROUGHT TOLERANCE AND BIOMASS ACCUMULATION THROUGH THE P...
- Publication number 20240150785
- Publication date May 9, 2024
- Peter K. Lundquist
- C07 - ORGANIC CHEMISTRY
Information Patent Application
COMPOSITIONS AND METHODS FOR TREATING CLRN1-ASSOCIATED HEARING LOSS...
- Publication number 20240139344
- Publication date May 2, 2024
- Akouos, Inc.
- Emmanuel John Simons
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
TARGETED GENE INSERTION FOR IMPROVED IMMUNE CELLS THERAPY
- Publication number 20240141293
- Publication date May 2, 2024
- CELLECTIS
- Brian BUSSER
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
METHODS AND COMPOSITIONS TO CONFER REGULATION TO GENE THERAPY CARGO...
- Publication number 20240141384
- Publication date May 2, 2024
- University of Florida Research Foundation, Incoporated
- Eric Tzy-Shi Wang
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
RECOMBINANT CONSTRUCTS AND TRANSGENIC FLUORESCENT ORNAMENTAL FISH T...
- Publication number 20240138383
- Publication date May 2, 2024
- GLOFISH LLC
- Alan BLAKE
- G06 - COMPUTING CALCULATING COUNTING
Information Patent Application
Viral Vectors For Treating Neurogenic Detrusor Overactivity
- Publication number 20240132897
- Publication date Apr 25, 2024
- Universite De Versailles-St Quentin En Yvelines
- François GIULIANO
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
ARTIFICIAL EXPRESSION CONSTRUCTS FOR MODULATING GENE EXPRESSION IN...
- Publication number 20240117377
- Publication date Apr 11, 2024
- Allen Institute
- Bryan Gore
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
GENE THERAPY FOR RETINAL DISEASES
- Publication number 20240102048
- Publication date Mar 28, 2024
- Greffex, Inc.
- Uwe D. STAERZ
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
FUNCTIONAL NUCLEIC ACID MOLECULE AND METHOD
- Publication number 20240092847
- Publication date Mar 21, 2024
- THE UNIVERSITY COURT OF THE UNIVERSITY OF EDINBURGH
- Steven Pollard
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
TREATMENT OF CONGENITAL STATIONARY NIGHT BLINDNESS USING GENE THERAPY
- Publication number 20240092858
- Publication date Mar 21, 2024
- SORBONNE UNIVERSITE
- Christina Zeitz
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
RAPID HIGH THROUGHPUT SCREENING SYSTEM TO ASSESS POTENTIAL TREATMEN...
- Publication number 20240093236
- Publication date Mar 21, 2024
- Trustees of Tufts College
- David L. Kaplan
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
COMPOSITIONS FOR AND METHODS OF IMPROVING FLUID FLUX IN THE BRAIN
- Publication number 20240082351
- Publication date Mar 14, 2024
- DUKE UNIVERSITY
- Aravind Asokan
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
METHOD OF DESIGNING HIGHLY REGULATED LENTIVIRAL VECTORS POSSESING S...
- Publication number 20240082427
- Publication date Mar 14, 2024
- The Regents of the University of California
- Donald B. Kohn
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
GLOBIN GENE THERAPY FOR TREATING HEMOGLOBINOPATHIES
- Publication number 20240066148
- Publication date Feb 29, 2024
- Memorial Sloan-Kettering Cancer Center
- Michel Sadelain
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
POLYNUCLEOTIDE FOR PHYSIOLOGICAL EXPRESSION IN T-CELLS
- Publication number 20240043868
- Publication date Feb 8, 2024
- FUNDACION PUBLICA ANDALUZA PROGRESO Y SALUD
- Francisco MARTÍN MOLINA
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
OPTIMIZED EXPRESSION CASSETTES FOR GENE THERAPY
- Publication number 20240042060
- Publication date Feb 8, 2024
- Tenaya Therapeutics, Inc.
- Huanyu ZHOU
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
COMPOSITIONS AND METHODS FOR TREATING BETA-HEMOGLOBINOPATHIES
- Publication number 20240043845
- Publication date Feb 8, 2024
- CSL Behring Gene Therapy, Inc.
- Jeffrey AHLERS
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
ARTIFICIAL EXPRESSION CONSTRUCTS FOR MODULATING GENE EXPRESSION IN...
- Publication number 20240018543
- Publication date Jan 18, 2024
- Allen Institute
- Tanya Daigle
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
Gene Expression System
- Publication number 20240016132
- Publication date Jan 18, 2024
- Oxitec Limited
- Martha KOUKIDOU
- A01 - AGRICULTURE FORESTRY ANIMAL HUSBANDRY HUNTING TRAPPING FISHING
Information Patent Application
ADENO-ASSOCIATED VIRUS (AAV) PRODUCER CELL LINE AND RELATED METHODS
- Publication number 20240018545
- Publication date Jan 18, 2024
- Lonza Walkersville, Inc.
- Bingnan GU
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
CRISPR-MEDIATED HUMAN GENOME EDITING WITH VECTORS
- Publication number 20230416776
- Publication date Dec 28, 2023
- Regents of the University of Minnesota
- Li Ou
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
TREATMENT OF HYPERBILIRUBINEMIA
- Publication number 20230414724
- Publication date Dec 28, 2023
- Genethon
- FEDERICO MINGOZZI
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
COMPOSITIONS AND METHODS FOR TREATING NON-AGE-ASSOCIATED HEARING IM...
- Publication number 20230407315
- Publication date Dec 21, 2023
- Akouos, Inc.
- Emmanuel John Simons
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
OPTIMIZED MINI-DYSTROPHIN GENES AND EXPRESSION CASSETTES AND THEIR USE
- Publication number 20230398236
- Publication date Dec 14, 2023
- The University of North Carolina at Chapel Hill
- Xiao Xiao
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
THERAPEUTIC ADENO-ASSOCIATED VIRUS DELIVERY OF FUKUTIN RELATED PROT...
- Publication number 20230374542
- Publication date Nov 23, 2023
- ASKLEPIOS BIOPHARMACEUTICAL, INC.
- Martin K. Childers
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
BIDIRECTIONAL DUAL PROMOTER EXPRESSION VECTORS AND USES THEREOF
- Publication number 20230374546
- Publication date Nov 23, 2023
- HOMOLOGY MEDICINES, INC.
- Carmen BARNES
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
CHIMERIC ANTIGEN RECEPTOR EXPRESSION SYSTEMS
- Publication number 20230364137
- Publication date Nov 16, 2023
- VOR BIOPHARMA INC.
- Sadik Kassim
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
METHODS FOR TREATING NEUROLOGICAL DISEASE
- Publication number 20230365963
- Publication date Nov 16, 2023
- ASKLEPIOS BIOPHARMACEUTICAL, INC.
- Anna TRETIAKOVA
- 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

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...

Expression cassette with a SynP160 promoter

Bicistronic AAV vector for RNA interference in ALS

Targeted gene insertion for improved immune cells therapy

SYNP161, a promoter for the expression of genes

Recombinant adenoviruses and stem cells comprising same

Lineage reporter synthetic chromosomes and methods of use

Methods of treating muscular dystrophy

Selective gene therapy expression system

Porcine Thy1 gene promoter specifically expressed in neurons

Lentiviral vector for treating hemoglobin disorders

Compositions and methods for treating beta-hemoglobinopathies

Expression vectors comprising engineered genes

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

Gene-therapy vectors for treating cardiomyopathy

Widespread gene delivery to motor neurons using peripheral injectio...

Globin gene therapy for treating hemoglobinopathies

High-transducing HSV vectors

Primate retinal pigment epithelium cell-specific promoter

Gene expression system

Patents Applicationslast 30 patents

PROMOTER FOR SPECIFIC EXPRESSION OF GENES IN ROD PHOTORECEPTORS

LIVER SPECIFIC PRODUCTION OF ENPP1 OR ENPP3

CONFERRING DROUGHT TOLERANCE AND BIOMASS ACCUMULATION THROUGH THE P...

COMPOSITIONS AND METHODS FOR TREATING CLRN1-ASSOCIATED HEARING LOSS...

TARGETED GENE INSERTION FOR IMPROVED IMMUNE CELLS THERAPY

METHODS AND COMPOSITIONS TO CONFER REGULATION TO GENE THERAPY CARGO...

RECOMBINANT CONSTRUCTS AND TRANSGENIC FLUORESCENT ORNAMENTAL FISH T...

Viral Vectors For Treating Neurogenic Detrusor Overactivity

ARTIFICIAL EXPRESSION CONSTRUCTS FOR MODULATING GENE EXPRESSION IN...

GENE THERAPY FOR RETINAL DISEASES

FUNCTIONAL NUCLEIC ACID MOLECULE AND METHOD

TREATMENT OF CONGENITAL STATIONARY NIGHT BLINDNESS USING GENE THERAPY

RAPID HIGH THROUGHPUT SCREENING SYSTEM TO ASSESS POTENTIAL TREATMEN...

COMPOSITIONS FOR AND METHODS OF IMPROVING FLUID FLUX IN THE BRAIN

METHOD OF DESIGNING HIGHLY REGULATED LENTIVIRAL VECTORS POSSESING S...

GLOBIN GENE THERAPY FOR TREATING HEMOGLOBINOPATHIES

POLYNUCLEOTIDE FOR PHYSIOLOGICAL EXPRESSION IN T-CELLS

OPTIMIZED EXPRESSION CASSETTES FOR GENE THERAPY

COMPOSITIONS AND METHODS FOR TREATING BETA-HEMOGLOBINOPATHIES

ARTIFICIAL EXPRESSION CONSTRUCTS FOR MODULATING GENE EXPRESSION IN...

Gene Expression System

ADENO-ASSOCIATED VIRUS (AAV) PRODUCER CELL LINE AND RELATED METHODS

CRISPR-MEDIATED HUMAN GENOME EDITING WITH VECTORS

TREATMENT OF HYPERBILIRUBINEMIA

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

OPTIMIZED MINI-DYSTROPHIN GENES AND EXPRESSION CASSETTES AND THEIR USE

THERAPEUTIC ADENO-ASSOCIATED VIRUS DELIVERY OF FUKUTIN RELATED PROT...

BIDIRECTIONAL DUAL PROMOTER EXPRESSION VECTORS AND USES THEREOF

CHIMERIC ANTIGEN RECEPTOR EXPRESSION SYSTEMS

METHODS FOR TREATING NEUROLOGICAL DISEASE