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A61K48/005

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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

A61K48/005characterised by an aspect of the 'active' part of the composition delivered

Industries

Patents Grantslast 30 patents

Information Patent Grant
Adeno-associated virus vector variants for high efficiency genome e...
- Patent number 11,965,174
- Issue date Apr 23, 2024
- City of Hope
- Saswati Chatterjee
- C07 - ORGANIC CHEMISTRY
Information Patent Grant
Targeted DNA demethylation method, fusion protein and application t...
- Patent number 11,965,187
- Issue date Apr 23, 2024
- GUANGZHOU MEDICAL UNIVERSITY
- Xi-Yong Yu
- C07 - ORGANIC CHEMISTRY
Information Patent Grant
Methods and compositions for genome editing in non-dividing cells
- Patent number 11,959,094
- Issue date Apr 16, 2024
- Salk Institute for Biological Studies
- Juan Carlos Izpisua Belmonte
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Gene therapy for tuberous sclerosis
- Patent number 11,958,887
- Issue date Apr 16, 2024
- The General Hospital Corporation
- Xandra Breakefield
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
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
Diagnostics and therapy for human respiratory syncytial virus
- Patent number 11,952,631
- Issue date Apr 9, 2024
- Medizinische Hochschule Hannover
- Daniel Todt
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Combination therapy for treatment of thoracic cancer using Ad-REIC/...
- Patent number 11,951,182
- Issue date Apr 9, 2024
- Momotaro-Gene Inc.
- Hiromi Kumon
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Compositions and methods for treating Huntington's disease
- Patent number 11,951,121
- Issue date Apr 9, 2024
- VOYAGER THERAPEUTICS, INC.
- Dinah Wen-Yee Sah
- 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
Biologically active compounds
- Patent number 11,944,688
- Issue date Apr 2, 2024
- SUTURA THERAPEUTICS LTD
- Keith Foster
- C07 - ORGANIC CHEMISTRY
Information Patent Grant
Engineered chimeric fusion protein compositions and methods of use...
- Patent number 11,944,680
- Issue date Apr 2, 2024
- Myeloid Therapeutics, Inc.
- Daniel Getts
- C07 - ORGANIC CHEMISTRY
Information Patent Grant
Compositions and therapeutic methods of microRNA gene delivery
- Patent number 11,946,064
- Issue date Apr 2, 2024
- The Brigham & Women's Hospital, Inc.
- Pierpaolo Peruzzi
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Modified cascade ribonucleoproteins and uses thereof
- Patent number 11,939,604
- Issue date Mar 26, 2024
- Caribou Biosciences, Inc.
- Stan Johan Jozef Brouns
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Compositions and methods for treating phenylketonuria
- Patent number 11,939,600
- Issue date Mar 26, 2024
- Arcturus Therapeutics, Inc.
- Kiyoshi Tachikawa
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Engraftable cell-based immunotherapy for long-term delivery of ther...
- Patent number 11,939,594
- Issue date Mar 26, 2024
- Seattle Children's Hospital
- David J. Rawlings
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Genetically modified MSC and therapeutic methods
- Patent number 11,939,591
- Issue date Mar 26, 2024
- The Regents of the University of California
- Jan Nolta
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Polynucleotides and vectors for the expression of transgenes
- Patent number 11,938,197
- Issue date Mar 26, 2024
- THE SYDNEY CHILDREN'S HOSPITALS NETWORK (RANDWICK AND WESTMEAD (INCORPORATING...
- Ian Alexander
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Compositions for treating heart disease by inhibiting the action of...
- Patent number 11,931,402
- Issue date Mar 19, 2024
- Anchored RSK3 Inhibitors, LLC
- Michael S. Kapiloff
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
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
Selective recovery
- Patent number 11,932,668
- Issue date Mar 19, 2024
- California Institute of Technology
- Benjamin E. Deverman
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Systemic delivery of adeno-associated virus vector expressing gamma...
- Patent number 11,925,675
- Issue date Mar 12, 2024
- Sarepta Therapeutics, Inc.
- Louise Rodino-Klapac
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Methods and materials for NT-3 gene therapy
- Patent number 11,926,653
- Issue date Mar 12, 2024
- Research Institute at Nationwide Children's Hospital
- Zarife Sahenk
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Compositions and methods for transient delivery of nucleases
- Patent number 11,920,168
- Issue date Mar 5, 2024
- University of Massachusetts
- Guangping Gao
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
RAAV-based compositions and methods
- Patent number 11,920,133
- Issue date Mar 5, 2024
- University of Massachusetts
- Terence Flotte
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Gene therapy for neurodegenerative disorders
- Patent number 11,911,440
- Issue date Feb 27, 2024
- Genzyme Corporation
- Marco A. Passini
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Hydrodynamic methods for delivering fluids to kidney tissues and re...
- Patent number 11,911,487
- Issue date Feb 27, 2024
- Indiana University Research and Technology Corporation
- Robert Bacallao
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Cardiac cell reprogramming with myocardin and ASCL1
- Patent number 11,913,012
- Issue date Feb 27, 2024
- Tenaya Therapeutics, Inc.
- Huanyu Zhou
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Methods for preparing and using highly active blood coagulation fac...
- Patent number 11,913,042
- Issue date Feb 27, 2024
- RUIJIN HOSPITAL SHANGHAI JIAOTONG UNIVERSITY SCHOOL OF MEDICINE
- Xuefeng Wang
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Non-transgene transfection for therapeutic purposes
- Patent number 11,912,744
- Issue date Feb 27, 2024
- Antibiotic Alternatives LLC
- Mark C. Herzberg
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Nucleic acid products and methods of administration thereof
- Patent number 11,904,023
- Issue date Feb 20, 2024
- Factor Bioscience Inc.
- Matthew Angel
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...

Patents Applicationslast 30 patents

Information Patent Application
METHODS AND COMPOSITIONS FOR REDUCING NUCLEIC ACID VECTOR-INDUCED T...
- Publication number 20240131190
- Publication date Apr 25, 2024
- Decibel Therapeutics, Inc.
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
RNA Containing Modified Nucleosides and Methods of Use Thereof
- Publication number 20240131194
- Publication date Apr 25, 2024
- The Trustees of the University of Pennsylvania
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
EFFECTOR PROTEINS, EFFECTOR PARTNERS, COMPOSITIONS, SYSTEMS AND MET...
- Publication number 20240131187
- Publication date Apr 25, 2024
- MAMMOTH BIOSCIENCES, INC.
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
COMPOSITIONS AND METHODS FOR ENHANCING GAMMA DELTA T CELLS IN THE GUT
- Publication number 20240131191
- Publication date Apr 25, 2024
- King's College London
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
MODIFIED POLYNUCLEOTIDES FOR THE PRODUCTION OF CYTOPLASMIC AND CYTO...
- Publication number 20240131193
- Publication date Apr 25, 2024
- Modema TX, Inc.
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
Compositions and Methods for Treatment of Ocular Disease Associated...
- Publication number 20240131195
- Publication date Apr 25, 2024
- 4D MOLECULAR THERAPEUTICS INC.
- 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
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
METHODS AND COMPOSITIONS FOR IN VIVO GENE EDITING BASED CELL-TYPE-S...
- Publication number 20240132861
- Publication date Apr 25, 2024
- California Institute of Technology
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
GENOME EDITING SYSTEMS COMPRISING REPAIR-MODULATING ENZYME MOLECULE...
- Publication number 20240132877
- Publication date Apr 25, 2024
- EDITAS MEDICINE, INC.
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
AAV TRANSFER CASSETTE
- Publication number 20240131185
- Publication date Apr 25, 2024
- StrideBio, Inc.
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
MODULATION OF GENE EXPRESSION FOR DISEASE TREATMENT
- Publication number 20240131188
- Publication date Apr 25, 2024
- Dogodan Therapeutics, Inc.
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
GENE THERAPY FOR TREM2-ASSOCIATED DISEASES AND DISORDERS
- Publication number 20240131192
- Publication date Apr 25, 2024
- Eli Lilly and Company
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
COMPOSITIONS AND METHODS FOR TREATING SENSORINEURAL HEARING LOSS US...
- Publication number 20240131186
- Publication date Apr 25, 2024
- Decibel Therapeutics, Inc.
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
VARIANT ADENO-ASSOCIATED VIRUS (AAV) CAPSID POLYPEPTIDES AND GENE T...
- Publication number 20240131189
- Publication date Apr 25, 2024
- Eli Lilly and Company
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
INDUCIBLE SINGLE AAV SYSTEM AND USES THEREOF
- Publication number 20240123086
- Publication date Apr 18, 2024
- University of Massachusetts
- Christian Mueller
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
COMPOSITIONS AND METHODS FOR THE TARGETING OF PCSK9
- Publication number 20240123089
- Publication date Apr 18, 2024
- Scribe Therapeutics Inc.
- Jason FERNANDES
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
CHIMERIC ANTIGEN RECEPTORS (CARs) TARGETING HEMATOLOGIC MALIGNANCIE...
- Publication number 20240124547
- Publication date Apr 18, 2024
- iCell Gene Therapeuticics LLC
- Yupo MA
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
COMPOUNDS AND COMPOSITIONS FOR INTRACELLULAR DELIVERY OF THERAPEUTI...
- Publication number 20240124388
- Publication date Apr 18, 2024
- ModernaTX, Inc.
- Kerry E. BENENATO
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
POLYNUCLEOTIDE COMPOSITIONS, RELATED FORMULATIONS, AND METHODS OF U...
- Publication number 20240123087
- Publication date Apr 18, 2024
- ReCode Therapeutics, Inc.
- Daniella Ishimaru
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
COMPOSITIONS AND METHODS FOR ENHANCING GAMMA DELTA T CELLS IN THE GUT
- Publication number 20240123090
- Publication date Apr 18, 2024
- King's College London
- Adrian HAYDAY
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
COMPOSITIONS AND METHODS FOR THE VECTORED AUGMENTATION OF PROTEIN D...
- Publication number 20240124889
- Publication date Apr 18, 2024
- Xiao-Qin Ren
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
VECTORIZED ANTI-CGRP AND ANTI-CGRPR ANTIBODIES AND ADMINISTRATION T...
- Publication number 20240124890
- Publication date Apr 18, 2024
- Regenxbio Inc.
- Jared Smith
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
OPTIMIZED FACTOR VIII GENES
- Publication number 20240124555
- Publication date Apr 18, 2024
- Bioverativ Therapeutics Inc.
- Siyuan TAN
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
MODIFIED NUCLEOSIDE, NUCLEOTIDE, AND NUCLEIC ACID COMPOSITIONS
- Publication number 20240123035
- Publication date Apr 18, 2024
- ModernaTX, Inc.
- Antonin DE FOUGEROLLES
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
LIVER SPECIFIC DELIVERY OF MESSENGER RNA
- Publication number 20240123084
- Publication date Apr 18, 2024
- Translate Bio, Inc.
- Braydon Charles Guild
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
AAV VECTORS TARGETING T-CELLS
- Publication number 20240123085
- Publication date Apr 18, 2024
- Ginkgo BioWorks, Inc.
- James Kennon Smith
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
USE OF A SPLIT dCAS FUSION PROTEIN SYSTEM FOR EPIGENETIC EDITING
- Publication number 20240123088
- Publication date Apr 18, 2024
- The Regents of the University of California
- Julian Halmai
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
COMPOSITIONS AND METHODS FOR TREATMENT OF NIEMANN PICK TYPE A DISEASE
- Publication number 20240115733
- Publication date Apr 11, 2024
- The Trustees of the University of Pennsylvania
- James M. WILSON
- 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...
Information Patent Application
MATERIALS AND METHODS FOR THE TREATMENT OF LYSOSOMAL ACID LIPASE DE...
- Publication number 20240115735
- Publication date Apr 11, 2024
- Research Institute at Nationwide Children's Hospital
- Paul Taylor Martin
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...

characterised by an aspect of the 'active' part of the composition delivered

Industry

Parent Industries

Current Industry

Patents Grantslast 30 patents

Adeno-associated virus vector variants for high efficiency genome e...

Targeted DNA demethylation method, fusion protein and application t...

Methods and compositions for genome editing in non-dividing cells

Gene therapy for tuberous sclerosis

Micro-dystrophins and related methods of use

Diagnostics and therapy for human respiratory syncytial virus

Combination therapy for treatment of thoracic cancer using Ad-REIC/...

Compositions and methods for treating Huntington's disease

Vector for gene silencing and replacement and methods of use thereof

Biologically active compounds

Engineered chimeric fusion protein compositions and methods of use...

Compositions and therapeutic methods of microRNA gene delivery

Modified cascade ribonucleoproteins and uses thereof

Compositions and methods for treating phenylketonuria

Engraftable cell-based immunotherapy for long-term delivery of ther...

Genetically modified MSC and therapeutic methods

Polynucleotides and vectors for the expression of transgenes

Compositions for treating heart disease by inhibiting the action of...

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

Selective recovery

Systemic delivery of adeno-associated virus vector expressing gamma...

Methods and materials for NT-3 gene therapy

Compositions and methods for transient delivery of nucleases

RAAV-based compositions and methods

Gene therapy for neurodegenerative disorders

Hydrodynamic methods for delivering fluids to kidney tissues and re...

Cardiac cell reprogramming with myocardin and ASCL1

Methods for preparing and using highly active blood coagulation fac...

Non-transgene transfection for therapeutic purposes

Nucleic acid products and methods of administration thereof

Patents Applicationslast 30 patents

METHODS AND COMPOSITIONS FOR REDUCING NUCLEIC ACID VECTOR-INDUCED T...

RNA Containing Modified Nucleosides and Methods of Use Thereof

EFFECTOR PROTEINS, EFFECTOR PARTNERS, COMPOSITIONS, SYSTEMS AND MET...

COMPOSITIONS AND METHODS FOR ENHANCING GAMMA DELTA T CELLS IN THE GUT

MODIFIED POLYNUCLEOTIDES FOR THE PRODUCTION OF CYTOPLASMIC AND CYTO...

Compositions and Methods for Treatment of Ocular Disease Associated...

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

METHODS AND COMPOSITIONS FOR IN VIVO GENE EDITING BASED CELL-TYPE-S...

GENOME EDITING SYSTEMS COMPRISING REPAIR-MODULATING ENZYME MOLECULE...

AAV TRANSFER CASSETTE

MODULATION OF GENE EXPRESSION FOR DISEASE TREATMENT

GENE THERAPY FOR TREM2-ASSOCIATED DISEASES AND DISORDERS

COMPOSITIONS AND METHODS FOR TREATING SENSORINEURAL HEARING LOSS US...

VARIANT ADENO-ASSOCIATED VIRUS (AAV) CAPSID POLYPEPTIDES AND GENE T...

INDUCIBLE SINGLE AAV SYSTEM AND USES THEREOF

COMPOSITIONS AND METHODS FOR THE TARGETING OF PCSK9

CHIMERIC ANTIGEN RECEPTORS (CARs) TARGETING HEMATOLOGIC MALIGNANCIE...

COMPOUNDS AND COMPOSITIONS FOR INTRACELLULAR DELIVERY OF THERAPEUTI...

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

COMPOSITIONS AND METHODS FOR ENHANCING GAMMA DELTA T CELLS IN THE GUT

COMPOSITIONS AND METHODS FOR THE VECTORED AUGMENTATION OF PROTEIN D...

VECTORIZED ANTI-CGRP AND ANTI-CGRPR ANTIBODIES AND ADMINISTRATION T...

OPTIMIZED FACTOR VIII GENES

MODIFIED NUCLEOSIDE, NUCLEOTIDE, AND NUCLEIC ACID COMPOSITIONS

LIVER SPECIFIC DELIVERY OF MESSENGER RNA

AAV VECTORS TARGETING T-CELLS

USE OF A SPLIT dCAS FUSION PROTEIN SYSTEM FOR EPIGENETIC EDITING

COMPOSITIONS AND METHODS FOR TREATMENT OF NIEMANN PICK TYPE A DISEASE

Method of Increasing the Function of an AAV Vector

MATERIALS AND METHODS FOR THE TREATMENT OF LYSOSOMAL ACID LIPASE DE...