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

Current Industry

A61K48/0058Nucleic acids adapted for tissue specific expression

Industries

Patents Grantslast 30 patents

Information Patent Grant
Means and methods for AAV gene therapy in humans
- Patent number 12,285,451
- Issue date Apr 29, 2025
- Uniqure IP B.V.
- Bart Antonius Nijmeijer
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Genome editing methods and constructs
- Patent number 12,285,495
- Issue date Apr 29, 2025
- FONDAZIONE TELETHON ETS
- Alberto Auricchio
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Adeno-associated virus virions for treatment of epilepsy
- Patent number 12,263,231
- Issue date Apr 1, 2025
- Jichi Medical University
- Shin-ichi Muramatsu
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Optimized mini-dystrophin genes and expression cassettes and their use
- Patent number 12,257,321
- Issue date Mar 25, 2025
- The University of North Carolina at Chapel Hill
- Xiao Xiao
- A01 - AGRICULTURE FORESTRY ANIMAL HUSBANDRY HUNTING TRAPPING FISHING
Information Patent Grant
Optimised coding sequence and promoter
- Patent number 12,257,319
- Issue date Mar 25, 2025
- UCL BUSINESS LTD
- Amit Nathwani
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Gene therapy for NMNAT1-associated retinal degeneration
- Patent number 12,257,320
- Issue date Mar 25, 2025
- Massachusetts Eye and Ear Infirmary
- Eric A. Pierce
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Methods of treating non-arteritic anterior ischemic optic neuropathy
- Patent number 12,252,518
- Issue date Mar 18, 2025
- Life Biosciences, Inc.
- Michel Wathier
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Myosin 15 promoters and uses thereof
- Patent number 12,252,520
- Issue date Mar 18, 2025
- Decibel Therapeutics, Inc.
- Joseph Burns
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Gene therapy for the treatment of CNGB1-linked retinitis pigmentosa
- Patent number 12,246,072
- Issue date Mar 11, 2025
- Stylianos Michalakis
- C07 - ORGANIC CHEMISTRY
Information Patent Grant
Treatment of mucopolysaccharidosis IVA
- Patent number 12,247,213
- Issue date Mar 11, 2025
- The Nemours Foundation
- Shunji Tomatsu
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Modified ligand-gated ion channels and methods of use
- Patent number 12,247,064
- Issue date Mar 11, 2025
- Howard Hughes Medical Institute
- Scott Sternson
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
DNA vector for targeted gene therapy
- Patent number 12,246,073
- Issue date Mar 11, 2025
- GENETIC DIAGNOSTICS AND THERAPY 21 LTD
- Anton Gamolski
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Adeno-associated virus factor VIII vectors
- Patent number 12,239,692
- Issue date Mar 4, 2025
- Biomarin Pharmaceutical Inc.
- Peter Cameron Colosi
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Drug and gene therapy to treat high myopia and other ocular disorde...
- Patent number 12,233,050
- Issue date Feb 25, 2025
- City University of Hong Kong
- Wenjun Xiong
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Therapeutic constructs for treating cancer
- Patent number 12,233,135
- Issue date Feb 25, 2025
- PRECISION MOLECULAR INC.
- Christopher Ullman
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Myosin 15 promoters and uses thereof
- Patent number 12,233,136
- Issue date Feb 25, 2025
- Decibel Therapeutics, Inc.
- Joseph Burns
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
GLP-1 and use thereof in compositions for treating metabolic diseases
- Patent number 12,221,615
- Issue date Feb 11, 2025
- The Trustees of the University of Pennsylvania
- Christian Hinderer
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Therapeutic applications of CPF1-based genome editing
- Patent number 12,214,056
- Issue date Feb 4, 2025
- Duke University
- Charles A. Gersbach
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Optimized human clotting factor IX gene expression cassettes and th...
- Patent number 12,209,263
- Issue date Jan 28, 2025
- The University of North Carolina at Chapel Hill
- Xiao Xiao
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Messenger RNA therapy for the treatment of ornithine transcarbamyla...
- Patent number 12,201,677
- Issue date Jan 21, 2025
- Translate Bio, Inc.
- Frank DeRosa
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Gene therapy for retinitis pigmentosa
- Patent number 12,201,698
- Issue date Jan 21, 2025
- Genzyme Corporation
- Catherine O'Riordan
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Adeno-associated virus compositions for restoring HBB gene function...
- Patent number 12,201,657
- Issue date Jan 21, 2025
- City of Hope
- Saswati Chatterjee
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
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...

Patents Applicationslast 30 patents

Information Patent Application
HYBRID REGULATORY ELEMENTS
- Publication number 20250127930
- Publication date Apr 24, 2025
- GENETHON
- FEDERICO MINGOZZI
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
TARGETED INTEGRATION AT BETA-GLOBIN LOCUS IN HUMAN HEMATOPOIETIC ST...
- Publication number 20250127927
- Publication date Apr 24, 2025
- The Board of Trustees of the Leland Stanford Junior University
- Matthew H. Porteus
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
SCAFFOLDS FOR MODIFYING IMMUNE CELLS AND THE USES THEREOF
- Publication number 20250127929
- Publication date Apr 24, 2025
- President and Fellows of Harvard College
- David T. Scadden
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
REGULATORY NUCLEIC ACID SEQUENCES
- Publication number 20250129381
- Publication date Apr 24, 2025
- ASKLEPIOS BIOPHARMACEUTICAL, INC.
- Jorge Omar YANEZ-CUNA
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
COMPOSITIONS AND PROCESSES FOR TARGETED DELIVERY, EXPRESSION AND MO...
- Publication number 20250129383
- Publication date Apr 24, 2025
- Combined Therapeutics, Inc.
- Romain Micol
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
GJB2 REGULATORY ELEMENTS AND USES THEREOF
- Publication number 20250127928
- Publication date Apr 24, 2025
- Decibel Therapeutics, Inc.
- Joseph BURNS
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
AAV PARTICLES WITH MODIFIED INVERTED TERMINAL REPEATS FOR ENHANCED...
- Publication number 20250121094
- Publication date Apr 17, 2025
- University of Florida Research Foundation, Incorporated
- Arun Srivastava
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
RECOMBINANT VIRUS EXPRESSING TPK AND USE THEREOF IN TREATMENT OF AL...
- Publication number 20250121095
- Publication date Apr 17, 2025
- SHANGHAI RAISING PHARMACEUTICAL CO., LTD.
- Jingjing WANG
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
TREATMENT OF RETINITIS PIGMENTOSA USING ENGINEERED MEGANUCLEASES
- Publication number 20250121039
- Publication date Apr 17, 2025
- PRECISION BIOSCIENCES, INC.
- Victor Bartsevich
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
INTRASPINAL DELIVERY OF THERAPEUTIC AGENTS
- Publication number 20250120997
- Publication date Apr 17, 2025
- The Trustees of the University of Pennsylvania
- Norbert Pardi
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
PROMOTERS, EXPRESSION CASSETTES, VECTORS, KITS, AND METHODS FOR THE...
- Publication number 20250121092
- Publication date Apr 17, 2025
- Beacon Therapeutics Limited
- Guo-jie Ye
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
ENGINEERED LIVER-SPECIFIC ENHANCERS AND THEIR APPLICATIONS
- Publication number 20250121096
- Publication date Apr 17, 2025
- Sichuan Real&Best Biotech Co., Ltd.
- Biao Dong
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
METHODS OF RECOMBINANT ADENO-ASSOCIATED VIRUS KIDNEY ADMINISTRATION
- Publication number 20250121097
- Publication date Apr 17, 2025
- ASKLEPIOS BIOPHARMACEUTICAL, INC.
- Philippe Moullier
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
THERAPEUTIC APPLICATIONS OF CPF1-BASED GENOME EDITING
- Publication number 20250114482
- Publication date Apr 10, 2025
- DUKE UNIVERSITY
- Charles A. Gersbach
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
VECTORS AND COMPOSITIONS FOR GENE AUGMENTATION OF CRUMBS COMPLEX HO...
- Publication number 20250108133
- Publication date Apr 3, 2025
- THE TRUSTEES OF COLUMBIA UNIVERSITY IN THE CITY OF NEW YORK
- Peter M.J. Quinn
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
SYSTEMIC ADMINISTRATION OF CIRCULAR RNA POLYNUCLEOTIDES ENCODING MU...
- Publication number 20250099621
- Publication date Mar 27, 2025
- Orna Therapeutics, Inc.
- Allen T. Horhota
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
AAV TRANSFER PLASMIDS
- Publication number 20250099617
- Publication date Mar 27, 2025
- Atsena Therapeutics, Inc.
- Christopher KOZLOWSKI
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
GENE THERAPY COMPOSITION AND TREATMENT FOR DYSTROPHIN-RELATED CARDI...
- Publication number 20250099620
- Publication date Mar 27, 2025
- DiNAQOR AG
- Valeria RICOTTI
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
COMPOSITIONS AND METHODS FOR EXPRESSING THERAPEUTICS
- Publication number 20250099622
- Publication date Mar 27, 2025
- Avirmax Biopharma Inc.
- Shengjiang LIU
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
GENERATION OF LARGE PROTEINS BY CO-DELIVERY OF MULTIPLE VECTORS
- Publication number 20250099616
- Publication date Mar 27, 2025
- University of Washington
- Jeffrey S. CHAMBERLAIN
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
DELIVERY AND USE OF THE CRISPR-CAS SYSTEMS, VECTORS AND COMPOSITION...
- Publication number 20250101468
- Publication date Mar 27, 2025
- THE BROAD INSTITUTE, INC.
- Feng ZHANG
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
MODIFIED ADENO-ASSOCIATED VIRUS VECTOR
- Publication number 20250099619
- Publication date Mar 27, 2025
- Juntendo Educational Foundation
- Kazusaku KAMIYA
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
METHODS AND COMPOSITIONS FOR THE TREATMENT OF RARE DISEASES
- Publication number 20250099624
- Publication date Mar 27, 2025
- Sangamo Therapeutics, Inc.
- Michael C. HOLMES
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
RECOMBINANT TERT-ENCODING VIRAL GENOMES AND VECTORS
- Publication number 20250099618
- Publication date Mar 27, 2025
- FUNDACIÓN DEL SECTOR PÚBLICO ESTATAL CENTRO NACIONAL DE INVESTIGACIONES
- María BLASCO
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
COMPOSITIONS AND METHODS FOR ENHANCING INTRA-MITOCHONDRIAL PROTEIN...
- Publication number 20250099623
- Publication date Mar 27, 2025
- THE BROAD INSTITUTE, INC.
- Vamsi MOOTHA
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
TREATMENT OF INTERVERTEBRAL DISC DEGENERATION
- Publication number 20250090635
- Publication date Mar 20, 2025
- Kolon TissueGene, Inc.
- Moon Jong Noh
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
ENGINEERED MUSCLE TARGETING COMPOSITIONS
- Publication number 20250090688
- Publication date Mar 20, 2025
- THE BROAD INSTITUTE, INC.
- Pardis SABETI
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
METHODS AND SYSTEMS FOR CORRECTING MUTATIONS IN PRPH2
- Publication number 20250090690
- Publication date Mar 20, 2025
- THE TRUSTEES OF COLUMBIA UNIVERSITY IN THE CITY OF NEW YORK
- Yi-Ting Tsai
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
EXPRESSION CASSETTES FOR TREATING EPILEPSY AND NEUROPATHIC PAIN
- Publication number 20250090689
- Publication date Mar 20, 2025
- TRAMES BIO, INC.
- Annahita Keravala
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
METHODS AND COMPOSITIONS RELATING TO THE TREATMENT OF TUMORS
- Publication number 20250082723
- Publication date Mar 13, 2025
- The General Hospital Corporation
- Gary J. BRENNER
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...

Nucleic acids adapted for tissue specific expression

Industry

Parent Industries

Current Industry

Patents Grantslast 30 patents

Means and methods for AAV gene therapy in humans

Genome editing methods and constructs

Adeno-associated virus virions for treatment of epilepsy

Optimized mini-dystrophin genes and expression cassettes and their use

Optimised coding sequence and promoter

Gene therapy for NMNAT1-associated retinal degeneration

Methods of treating non-arteritic anterior ischemic optic neuropathy

Myosin 15 promoters and uses thereof

Gene therapy for the treatment of CNGB1-linked retinitis pigmentosa

Treatment of mucopolysaccharidosis IVA

Modified ligand-gated ion channels and methods of use

DNA vector for targeted gene therapy

Adeno-associated virus factor VIII vectors

Drug and gene therapy to treat high myopia and other ocular disorde...

Therapeutic constructs for treating cancer

Myosin 15 promoters and uses thereof

GLP-1 and use thereof in compositions for treating metabolic diseases

Therapeutic applications of CPF1-based genome editing

Optimized human clotting factor IX gene expression cassettes and th...

Messenger RNA therapy for the treatment of ornithine transcarbamyla...

Gene therapy for retinitis pigmentosa

Adeno-associated virus compositions for restoring HBB gene function...

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...

Patents Applicationslast 30 patents

HYBRID REGULATORY ELEMENTS

TARGETED INTEGRATION AT BETA-GLOBIN LOCUS IN HUMAN HEMATOPOIETIC ST...

SCAFFOLDS FOR MODIFYING IMMUNE CELLS AND THE USES THEREOF

REGULATORY NUCLEIC ACID SEQUENCES

COMPOSITIONS AND PROCESSES FOR TARGETED DELIVERY, EXPRESSION AND MO...

GJB2 REGULATORY ELEMENTS AND USES THEREOF

AAV PARTICLES WITH MODIFIED INVERTED TERMINAL REPEATS FOR ENHANCED...

RECOMBINANT VIRUS EXPRESSING TPK AND USE THEREOF IN TREATMENT OF AL...

TREATMENT OF RETINITIS PIGMENTOSA USING ENGINEERED MEGANUCLEASES

INTRASPINAL DELIVERY OF THERAPEUTIC AGENTS

PROMOTERS, EXPRESSION CASSETTES, VECTORS, KITS, AND METHODS FOR THE...

ENGINEERED LIVER-SPECIFIC ENHANCERS AND THEIR APPLICATIONS

METHODS OF RECOMBINANT ADENO-ASSOCIATED VIRUS KIDNEY ADMINISTRATION

THERAPEUTIC APPLICATIONS OF CPF1-BASED GENOME EDITING

VECTORS AND COMPOSITIONS FOR GENE AUGMENTATION OF CRUMBS COMPLEX HO...

SYSTEMIC ADMINISTRATION OF CIRCULAR RNA POLYNUCLEOTIDES ENCODING MU...

AAV TRANSFER PLASMIDS

GENE THERAPY COMPOSITION AND TREATMENT FOR DYSTROPHIN-RELATED CARDI...

COMPOSITIONS AND METHODS FOR EXPRESSING THERAPEUTICS

GENERATION OF LARGE PROTEINS BY CO-DELIVERY OF MULTIPLE VECTORS

DELIVERY AND USE OF THE CRISPR-CAS SYSTEMS, VECTORS AND COMPOSITION...

MODIFIED ADENO-ASSOCIATED VIRUS VECTOR

METHODS AND COMPOSITIONS FOR THE TREATMENT OF RARE DISEASES

RECOMBINANT TERT-ENCODING VIRAL GENOMES AND VECTORS

COMPOSITIONS AND METHODS FOR ENHANCING INTRA-MITOCHONDRIAL PROTEIN...

TREATMENT OF INTERVERTEBRAL DISC DEGENERATION

ENGINEERED MUSCLE TARGETING COMPOSITIONS

METHODS AND SYSTEMS FOR CORRECTING MUTATIONS IN PRPH2

EXPRESSION CASSETTES FOR TREATING EPILEPSY AND NEUROPATHIC PAIN

METHODS AND COMPOSITIONS RELATING TO THE TREATMENT OF TUMORS