Purification or manufacturing processes for gene therapy compositions

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A61K48/0091

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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

A61K48/0091Purification or manufacturing processes for gene therapy compositions

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Patents Grantslast 30 patents

Information Patent Grant
Smart multidosing
- Patent number 12,293,815
- Issue date May 6, 2025
- OptimDosing LLC
- David Inwald
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Allele-specific inactivation of mutant HTT via gene editing at codi...
- Patent number 12,268,755
- Issue date Apr 8, 2025
- UNIVERSITY OF MASSACHUSETTS
- Michael Harry Brodsky
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Compositions and methods for transient gene therapy with enhanced s...
- Patent number 12,258,560
- Issue date Mar 25, 2025
- Dana-Farber Cancer Institute, Inc.
- Michael Goldberg
- C07 - ORGANIC CHEMISTRY
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
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
Dry powder composition comprising long-chain RNA
- Patent number 12,208,165
- Issue date Jan 28, 2025
- CureVac SE
- Fabian Johannes Eber
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Compositions and methods for promoting homology directed repair
- Patent number 12,201,699
- Issue date Jan 21, 2025
- Editas Medicine, Inc.
- Cecilia Cotta-Ramusino
- 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
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
Dry powder composition comprising long-chain RNA
- Patent number 12,138,348
- Issue date Nov 12, 2024
- CureVac SE
- Fabian Johannes Eber
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
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
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
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
Gene therapy
- Patent number 12,059,478
- Issue date Aug 13, 2024
- Ospedale San Raffaele S.r.l.
- Anna Christina Kajaste-Rudnitski
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Treating metastatic cancer and model systems for metastatic disease
- Patent number 12,059,480
- Issue date Aug 13, 2024
- Memorial Sloan Kettering Cancer Center
- Karuna Ganesh
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Nanoparticles comprising protein-polynucleotide complexes and for d...
- Patent number 12,036,287
- Issue date Jul 16, 2024
- Asklepios Biopharmaceutical, Inc.
- Gretchen M. Unger
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Extracellular vesicles for inhalation
- Patent number 12,029,821
- Issue date Jul 9, 2024
- OMNISPIRANT LIMITED
- Gerard Bernard McCauley
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Rapid design, build, test, and learn technologies for identifying a...
- Patent number 12,031,128
- Issue date Jul 9, 2024
- Battelle Memorial Institute
- Anthony D. Duong
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Delivery and use of the CRISPR-CAS systems, vectors and composition...
- Patent number 12,018,275
- Issue date Jun 25, 2024
- The Broad Institute, Inc.
- Feng Zhang
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Compositions and systems comprising transfection-competent vesicles...
- Patent number 11,980,673
- Issue date May 14, 2024
- The University of British Columbia
- Blair Leavitt
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Processes of preparing mRNA-loaded lipid nanoparticles
- Patent number 11,969,480
- Issue date Apr 30, 2024
- Translate Bio, Inc.
- Shrirang Karve
- B01 - PHYSICAL OR CHEMICAL PROCESSES OR APPARATUS IN GENERAL
Information Patent Grant
Nucleic acid loaded extracellular vesicles
- Patent number 11,970,718
- Issue date Apr 30, 2024
- Carmine Therapeutics Pte. Ltd.
- Harvey Lodish
- B82 - NANO-TECHNOLOGY
Information Patent Grant
Compositions and methods for TTR gene editing and treating ATTR amy...
- Patent number 11,965,165
- Issue date Apr 23, 2024
- Intellia Therapeutics, Inc.
- Arti Mahendra Prakash Kanjolia
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Lipid nanoparticle compositions and methods for MRNA delivery
- Patent number 11,951,180
- Issue date Apr 9, 2024
- Translate Bio, Inc.
- Frank DeRosa
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Lipid nanoparticle compositions and methods for mRNA delivery
- Patent number 11,951,181
- Issue date Apr 9, 2024
- Translate Bio, Inc.
- Frank DeRosa
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Lipid nanoparticle compositions and methods for MRNA delivery
- Patent number 11,951,179
- Issue date Apr 9, 2024
- Translate Bio, Inc.
- Frank DeRosa
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Vector for gene silencing and replacement and methods of use thereof
- Patent number 11,951,184
- Issue date Apr 9, 2024
- Wisconsin Alumni Research Foundation
- Matthew Michael Millette
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Evolution of TALENs
- Patent number 11,913,040
- Issue date Feb 27, 2024
- President and Fellows of Harvard College
- David R. Liu
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Scalable manufacturing platform for viral vector purification and v...
- Patent number 11,878,056
- Issue date Jan 23, 2024
- The Children's Hospital of Philadelphia
- John Fraser Wright
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
RAAV with chemically modified capsid
- Patent number 11,865,185
- Issue date Jan 9, 2024
- Centre National de la Recherche Scientifique (Cnrs)
- Mathieu Mevel
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...

Patents Applicationslast 30 patents

Information Patent Application
METHOD OF INCREASING THE FUNCTION OF AN AAV VECTOR
- Publication number 20250171801
- Publication date May 29, 2025
- The Trustees of the University of Pennsylvania
- Luk VANDENBERGHE
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
Lipid Nanoparticle Compositions and Methods for mRNA Delivery
- Publication number 20250152735
- Publication date May 15, 2025
- Translate Bio, Inc.
- Frank DeRosa
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
NOVEL IMMUNOSTIMULATING VECTOR SYSTEM
- Publication number 20250127931
- Publication date Apr 24, 2025
- Provecs Medical GmbH
- Frank Schnieders
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
METHOD FOR DELIVERING RNA TO NEURONS TO TREAT HERPES INFECTIONS
- Publication number 20250127932
- Publication date Apr 24, 2025
- University of Florida Research Foundation, Incorporated
- David C. Bloom
- 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
DRY POWDER COMPOSITION COMPRISING LONG-CHAIN RNA
- Publication number 20250114307
- Publication date Apr 10, 2025
- CureVac SE
- Fabian Johannes EBER
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
Lipid Nanoparticle Compositions and Methods for mRNA Delivery
- Publication number 20250108126
- Publication date Apr 3, 2025
- Translate Bio, Inc.
- Frank DeRosa
- 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
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
STABILIZED FORMULATIONS OF CHIMPANZEE ADENOVIRUS
- Publication number 20250101461
- Publication date Mar 27, 2025
- Gritstone bio, Inc.
- Arunendra Saha Ray
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
GENE EDITING OF CAR-T CELLS FOR THE TREATMENT OF T CELL MALIGNANCIE...
- Publication number 20250082785
- Publication date Mar 13, 2025
- Washington University
- John F. DiPersio
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
GENE THERAPY FOR RECESSIVE DYSTROPHIC EPIDERMOLYSIS BULLOSA USING G...
- Publication number 20250075178
- Publication date Mar 6, 2025
- The Board of Trustees of the Leland Stanford Junior University
- Zurab Siprashvili
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
CELL-TYPE SPECIFIC MEMBRANE FUSION PROTEINS
- Publication number 20250057984
- Publication date Feb 20, 2025
- THE BROAD INSTITUTE, INC.
- Feng ZHANG
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
METHODS FOR FORMING POLYPLEXES
- Publication number 20250041458
- Publication date Feb 6, 2025
- AMRYT GENETICS LTD.
- Conall O'Broin
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
PROCESSES OF PREPARING MRNA-LOADED LIPID NANOPARTICLES
- Publication number 20240366798
- Publication date Nov 7, 2024
- Translate Bio, Inc.
- Shrirang Karve
- B01 - PHYSICAL OR CHEMICAL PROCESSES OR APPARATUS IN GENERAL
Information Patent Application
COMPOSITIONS AND SYSTEMS COMPRISING TRANSFECTION-COMPETENT VESICLES...
- Publication number 20240350676
- Publication date Oct 24, 2024
- The University of British Columbia
- Blair Leavitt
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
COMPOSITIONS AND SYSTEMS COMPRISING TRANSFECTION-COMPETENT VESICLES...
- Publication number 20240342315
- Publication date Oct 17, 2024
- The University of British Columbia
- Blair Leavitt
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
EXTRACELLULAR VESICLES FOR INHALATION
- Publication number 20240315980
- Publication date Sep 26, 2024
- OMNISPIRANT LIMITED
- Gerard Bernard MCCAULEY
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
CAPSID VARIANTS AND METHODS OF USING THE SAME
- Publication number 20240294578
- Publication date Sep 5, 2024
- DYNO THERAPEUTICS, INC.
- Sylvain Lapan
- C07 - ORGANIC CHEMISTRY
Information Patent Application
SCALABLE PRODUCTION METHOD FOR AAV
- Publication number 20240247237
- Publication date Jul 25, 2024
- The Trustees of the University of Pennsylvania
- Martin Lock
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
CONSTRUCTS AND METHODS FOR DELIVERYING MOLECULES VIA VIRAL VECTORS...
- Publication number 20240229068
- Publication date Jul 11, 2024
- NXGEN VECTOR SOLUTIONS, LLC
- Joseph E. Rabinowitz
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
ELECTROPORATION, DEVELOPMENTALLY-ACTIVATED CELLS, PLURIPOTENT-LIKE...
- Publication number 20240228941
- Publication date Jul 11, 2024
- Christopher B. Reid
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
EVOLUTION OF TALENS
- Publication number 20240200044
- Publication date Jun 20, 2024
- President and Fellows of Harvard College
- David R. Liu
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
HSV AMPLICON PACKAGING SYSTEM USING ENGINEERED CELLS
- Publication number 20240191243
- Publication date Jun 13, 2024
- University of Pittsburgh of the Commonwealth System of Higher Education
- Joseph C. Glorioso
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
PRODUCTION OF CIRCULAR POLYRIBONUCLEOTIDES IN A EUKARYOTIC SYSTEM
- Publication number 20240181079
- Publication date Jun 6, 2024
- Flagship Pioneering Innovations VII, LLC
- Barry Andrew Martin
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
METHODS OF PREPARATION OF NOVEL PAN TLR ANTAGONISTIC LIPOSOMAL-LNP...
- Publication number 20240156733
- Publication date May 16, 2024
- Celestial Therapeutics Inc.
- Ajay Gupta
- B82 - NANO-TECHNOLOGY
Information Patent Application
Methods and Compositions for Modifying a Mutant Dystrophin Gene in...
- Publication number 20240156989
- Publication date May 16, 2024
- The Regents of the University of California
- April D. Pyle
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
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
CONSTRUCTS AND METHODS FOR DELIVERYING MOLECULES VIA VIRAL VECTORS...
- Publication number 20240132912
- Publication date Apr 25, 2024
- NXGEN VECTOR SOLUTIONS, LLC
- Joseph E. Rabinowitz
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
Method of Increasing the Function of an AAV Vector
- Publication number 20240117378
- Publication date Apr 11, 2024
- The Trustees of the University of Pennsylvania
- Luk VANDENBERGHE
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...

Purification or manufacturing processes for gene therapy compositions

Industry

Parent Industries

Current Industry

Patents Grantslast 30 patents

Smart multidosing

Allele-specific inactivation of mutant HTT via gene editing at codi...

Compositions and methods for transient gene therapy with enhanced s...

Modified ligand-gated ion channels and methods of use

Therapeutic applications of CPF1-based genome editing

Dry powder composition comprising long-chain RNA

Compositions and methods for promoting homology directed repair

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

Method for delivering RNA to neurons to treat herpes infections

Dry powder composition comprising long-chain RNA

Immunostimulating vector system

Lipid nanoparticle compositions and methods for mRNA delivery

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

Gene therapy

Treating metastatic cancer and model systems for metastatic disease

Nanoparticles comprising protein-polynucleotide complexes and for d...

Extracellular vesicles for inhalation

Rapid design, build, test, and learn technologies for identifying a...

Delivery and use of the CRISPR-CAS systems, vectors and composition...

Compositions and systems comprising transfection-competent vesicles...

Processes of preparing mRNA-loaded lipid nanoparticles

Nucleic acid loaded extracellular vesicles

Compositions and methods for TTR gene editing and treating ATTR amy...

Lipid nanoparticle compositions and methods for MRNA delivery

Lipid nanoparticle compositions and methods for mRNA delivery

Lipid nanoparticle compositions and methods for MRNA delivery

Vector for gene silencing and replacement and methods of use thereof

Evolution of TALENs

Scalable manufacturing platform for viral vector purification and v...

RAAV with chemically modified capsid

Patents Applicationslast 30 patents

METHOD OF INCREASING THE FUNCTION OF AN AAV VECTOR

Lipid Nanoparticle Compositions and Methods for mRNA Delivery

NOVEL IMMUNOSTIMULATING VECTOR SYSTEM

METHOD FOR DELIVERING RNA TO NEURONS TO TREAT HERPES INFECTIONS

THERAPEUTIC APPLICATIONS OF CPF1-BASED GENOME EDITING

DRY POWDER COMPOSITION COMPRISING LONG-CHAIN RNA

Lipid Nanoparticle Compositions and Methods for mRNA Delivery

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

METHODS AND COMPOSITIONS FOR THE TREATMENT OF RARE DISEASES

STABILIZED FORMULATIONS OF CHIMPANZEE ADENOVIRUS

GENE EDITING OF CAR-T CELLS FOR THE TREATMENT OF T CELL MALIGNANCIE...

GENE THERAPY FOR RECESSIVE DYSTROPHIC EPIDERMOLYSIS BULLOSA USING G...

CELL-TYPE SPECIFIC MEMBRANE FUSION PROTEINS

METHODS FOR FORMING POLYPLEXES

PROCESSES OF PREPARING MRNA-LOADED LIPID NANOPARTICLES

COMPOSITIONS AND SYSTEMS COMPRISING TRANSFECTION-COMPETENT VESICLES...

COMPOSITIONS AND SYSTEMS COMPRISING TRANSFECTION-COMPETENT VESICLES...

EXTRACELLULAR VESICLES FOR INHALATION

CAPSID VARIANTS AND METHODS OF USING THE SAME

SCALABLE PRODUCTION METHOD FOR AAV

CONSTRUCTS AND METHODS FOR DELIVERYING MOLECULES VIA VIRAL VECTORS...

ELECTROPORATION, DEVELOPMENTALLY-ACTIVATED CELLS, PLURIPOTENT-LIKE...

EVOLUTION OF TALENS

HSV AMPLICON PACKAGING SYSTEM USING ENGINEERED CELLS

PRODUCTION OF CIRCULAR POLYRIBONUCLEOTIDES IN A EUKARYOTIC SYSTEM

METHODS OF PREPARATION OF NOVEL PAN TLR ANTAGONISTIC LIPOSOMAL-LNP...

Methods and Compositions for Modifying a Mutant Dystrophin Gene in...

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

CONSTRUCTS AND METHODS FOR DELIVERYING MOLECULES VIA VIRAL VECTORS...

Method of Increasing the Function of an AAV Vector