Manipulation of the nucleic acid to modify its expression pattern

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A61K48/0066

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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

A61K48/0066Manipulation of the nucleic acid to modify its expression pattern

Industries

Patents Grantslast 30 patents

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
RNA constructs and uses thereof
- Patent number 11,951,185
- Issue date Apr 9, 2024
- BIONTECH SE
- Ugur Sahin
- B82 - NANO-TECHNOLOGY
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
Adeno-associated virus compositions for PAH gene transfer and metho...
- Patent number 11,951,183
- Issue date Apr 9, 2024
- HOMOLOGY MEDICINES, INC.
- Albert Barnes Seymour
- 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
Treatment of heart disease by disruption of the anchoring of PP2A
- Patent number 11,938,198
- Issue date Mar 26, 2024
- University of Miami
- Michael S. Kapiloff
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Recombinogenic nucleic acid strands in situ
- Patent number 11,931,426
- Issue date Mar 19, 2024
- SNIPR TECHNOLOGIES LIMITED
- Jasper Clube
- 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
Coronavirus vaccine
- Patent number 11,925,694
- Issue date Mar 12, 2024
- BIONTECH SE
- Ugur Sahin
- B82 - NANO-TECHNOLOGY
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
Viral vectors for cancer therapy
- Patent number 11,918,660
- Issue date Mar 5, 2024
- Krystal Biotech, Inc.
- Suma Krishnan
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
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
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...
Information Patent Grant
Compositions and methods for the expression of CRISPR guide RNAs us...
- Patent number 11,896,679
- Issue date Feb 13, 2024
- The Johns Hopkins University
- Vinod Jaskula-Ranga
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Methods for regulating endogenous production of lactoferrin and sub...
- Patent number 11,891,429
- Issue date Feb 6, 2024
- Cellastra Inc.
- Bradley G. Thompson
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Enhancing health in mammals using telomerase reverse transcriptase...
- Patent number 11,883,471
- Issue date Jan 30, 2024
- Sierra Sciences, LLC
- William H. Andrews
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Electroporation, developmentally-activated cells, pluripotent-like...
- Patent number 11,859,168
- Issue date Jan 2, 2024
- Christopher B. Reid
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Compositions and methods for the expression of CRISPR guide RNAs us...
- Patent number 11,850,289
- Issue date Dec 26, 2023
- The Johns Hopkins University
- Vinod Jaskula-Ranga
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
HSD17B13 variants and uses thereof
- Patent number 11,845,963
- Issue date Dec 19, 2023
- REGENERON PHARMACEUTICALS, INC.
- Noura S. Abul-Husn
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Therapeutic editing
- Patent number 11,827,880
- Issue date Nov 28, 2023
- Shape Therapeutics Inc.
- David Huss
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Targeting chromosomal instability and downstream cytosolic DNA sign...
- Patent number 11,821,042
- Issue date Nov 21, 2023
- Cornell University
- Lewis C. Cantley
- 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
PAR1 modulation to alter myelination
- Patent number 11,813,264
- Issue date Nov 14, 2023
- Mayo Foundation for Medical Education and Research
- Isobel A Scarisbrick
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Codon optimized GLA genes and uses thereof
- Patent number 11,802,278
- Issue date Oct 31, 2023
- David H. Kirn
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Materials and methods for treatment of pain related disorders
- Patent number 11,801,313
- Issue date Oct 31, 2023
- Vertex Pharmaceuticals Incorporated
- Ante Sven Lundberg
- C07 - ORGANIC CHEMISTRY
Information Patent Grant
RNA containing modified nucleosides and methods of use thereof
- Patent number 11,801,314
- Issue date Oct 31, 2023
- The Trustees of the University of Pennsylvania
- Katalin Kariko
- C07 - ORGANIC CHEMISTRY
Information Patent Grant
Methods for selective kinase inhibition by endogenously produced an...
- Patent number 11,793,889
- Issue date Oct 24, 2023
- KINASE PHARMA INC.
- Bradley G Thompson
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
RNA cleavage-induced transcript stabilizer and uses thereof
- Patent number 11,795,455
- Issue date Oct 24, 2023
- Massachusetts Institute of Technology
- Ron Weiss
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
ELOVL2 constructs for human gene therapy
- Patent number 11,793,890
- Issue date Oct 24, 2023
- Visgenx, Inc.
- Christopher Chavez
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Methods for treating VEGF-related conditions
- Patent number 11,786,611
- Issue date Oct 17, 2023
- Murdoch University
- Rakesh Veedu
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE

Patents Applicationslast 30 patents

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
- Katalin Kariko
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
Systems and Methods to Enhance RNA Stability and Translation and Us...
- Publication number 20240131196
- Publication date Apr 25, 2024
- The Board of Trustees of the Leland Stanford Junior University
- Rhiju Das
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
Compositions For and Methods of Improving Gene Therapy
- Publication number 20240131197
- Publication date Apr 25, 2024
- DUKE UNIVERSITY
- Aravind Asokan
- 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.
- Christian BURNS
- 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.
- Antonin DE FOUGEROLLES
- 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
- Steven Pennock
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
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
RNA CLEAVAGE-INDUCED TRANSCRIPT STABILIZER AND USES THEREOF
- Publication number 20240124877
- Publication date Apr 18, 2024
- Massachusetts Institute of Technology
- Ron Weiss
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
OLIGONUCLEOTIDE COMPOSITIONS AND METHODS THEREOF
- Publication number 20240117347
- Publication date Apr 11, 2024
- WAVE LIFE SCIENCES LTD.
- David Charles Donnell Butler
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
TARGETING CHROMOSOMAL INSTABILITY AND DOWNSTREAM CYTOSOLIC DNA SIGN...
- Publication number 20240110246
- Publication date Apr 4, 2024
- Cornell University
- Lewis C. Cantley
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
PROGRAMMABLE RNA WRITING USING CRISPR EFFECTORS AND TRANS-SPLICING...
- Publication number 20240100192
- Publication date Mar 28, 2024
- Massachusetts Institute of Technology
- Omar Osama Abudayyeh
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
Antibody Delivery
- Publication number 20240100187
- Publication date Mar 28, 2024
- AC IMMUNE SA
- Gregorio Del Val
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
Brain Disease Therapy Enhancing Inhibition of GABAergic Neurons
- Publication number 20240100190
- Publication date Mar 28, 2024
- City University of Hong Kong
- Jufang He
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
METHODS AND COMPOSITIONS FOR MODULATING GENE EXPRESSION
- Publication number 20240100191
- Publication date Mar 28, 2024
- Wisconsin Alumni Research Foundation
- Aseem Ansari
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
Self-complementary AAV vectors carrying dominant negative RhoA and...
- Publication number 20240100193
- Publication date Mar 28, 2024
- IVIEW Therapeutics, Inc.
- Terete Borras
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
USING MINIVECTORS TO TREAT IDIOPATHIC PULMONARY FIBROSIS
- Publication number 20240100189
- Publication date Mar 28, 2024
- Twister Biotech, Inc
- E. Lynn ZECHIEDRICH
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
IMPORTATION OF MITOCHONDRIAL PROTEIN BY AN ENHANCED ALLOTOPIC APPROACH
- Publication number 20240093229
- Publication date Mar 21, 2024
- INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (I.N.S.E.R.M)
- Marisol Corral-Debrinski
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
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
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
TELOMERASE REVERSE TRANSCRIPTASE-BASED THERAPIES
- Publication number 20240075109
- Publication date Mar 7, 2024
- Fundación del Sector Público Estatal Centro Nacional de Investigaciones Oncol...
- Maria Bobadilla
- A01 - AGRICULTURE FORESTRY ANIMAL HUSBANDRY HUNTING TRAPPING FISHING
Information Patent Application
CORONAVIRUS VACCINE
- Publication number 20240075165
- Publication date Mar 7, 2024
- BIONTECH SE
- Ugur Sahin
- B82 - NANO-TECHNOLOGY
Information Patent Application
VIRAL VECTORS AND NUCLEIC ACIDS FOR REGULATED GENE THERAPY
- Publication number 20240076689
- Publication date Mar 7, 2024
- Boehringer Ingelheim International GmbH
- John Park
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
METHODS AND COMPOSITIONS FOR TREATING TUMOR CELLS
- Publication number 20240075166
- Publication date Mar 7, 2024
- Stitch Bio, LLC
- Anthony P. Shuber
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
AAV-MEDIATED EXPRESSION USING A SYNTHETIC PROMOTER AND ENHANCER
- Publication number 20240066147
- Publication date Feb 29, 2024
- University of Iowa Research Foundation
- John F. Engelhardt
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
MATERIALS AND METHODS FOR TREATMENT OF PAIN RELATED DISORDERS
- Publication number 20240066150
- Publication date Feb 29, 2024
- Vertex Phamaceuticals Incorporated
- Ante Sven Lundberg
- 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
Nucleic Acid Molecule Containing Incorporated Gene Encoding Fused P...
- Publication number 20240066149
- Publication date Feb 29, 2024
- JCR PHARMACEUTICALS CO., LTD
- Masafumi Kinoshita
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
METHODS OF TREATING EYE DISEASES
- Publication number 20240066151
- Publication date Feb 29, 2024
- Visgenx, Inc.
- Christopher Chavez
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
METHODS AND COMPOSITIONS FOR TREATMENT OF FRIEDREICH'S ATAXIA
- Publication number 20240058477
- Publication date Feb 22, 2024
- University of Florida Research Foundation, Incorporated
- Manuela Corti
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
METHODS AND COMPOSITIONS FOR TREATING A PREMATURE STOP CODON-MEDIAT...
- Publication number 20240050594
- Publication date Feb 15, 2024
- Case Western Reserve University
- Jeffery M. Coller
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE

Manipulation of the nucleic acid to modify its expression pattern

Industry

Parent Industries

Current Industry

Patents Grantslast 30 patents

Methods and compositions for genome editing in non-dividing cells

RNA constructs and uses thereof

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

Adeno-associated virus compositions for PAH gene transfer and metho...

Vector for gene silencing and replacement and methods of use thereof

Treatment of heart disease by disruption of the anchoring of PP2A

Recombinogenic nucleic acid strands in situ

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

Coronavirus vaccine

Engineered muscle targeting compositions

Viral vectors for cancer therapy

Evolution of TALENs

Nucleic acid products and methods of administration thereof

Compositions and methods for the expression of CRISPR guide RNAs us...

Methods for regulating endogenous production of lactoferrin and sub...

Enhancing health in mammals using telomerase reverse transcriptase...

Electroporation, developmentally-activated cells, pluripotent-like...

Compositions and methods for the expression of CRISPR guide RNAs us...

HSD17B13 variants and uses thereof

Therapeutic editing

Targeting chromosomal instability and downstream cytosolic DNA sign...

Selective gene therapy expression system

PAR1 modulation to alter myelination

Codon optimized GLA genes and uses thereof

Materials and methods for treatment of pain related disorders

RNA containing modified nucleosides and methods of use thereof

Methods for selective kinase inhibition by endogenously produced an...

RNA cleavage-induced transcript stabilizer and uses thereof

ELOVL2 constructs for human gene therapy

Methods for treating VEGF-related conditions

Patents Applicationslast 30 patents

RNA Containing Modified Nucleosides and Methods of Use Thereof

Systems and Methods to Enhance RNA Stability and Translation and Us...

Compositions For and Methods of Improving Gene Therapy

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

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

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

MODIFIED NUCLEOSIDE, NUCLEOTIDE, AND NUCLEIC ACID COMPOSITIONS

RNA CLEAVAGE-INDUCED TRANSCRIPT STABILIZER AND USES THEREOF

OLIGONUCLEOTIDE COMPOSITIONS AND METHODS THEREOF

TARGETING CHROMOSOMAL INSTABILITY AND DOWNSTREAM CYTOSOLIC DNA SIGN...

PROGRAMMABLE RNA WRITING USING CRISPR EFFECTORS AND TRANS-SPLICING...

Antibody Delivery

Brain Disease Therapy Enhancing Inhibition of GABAergic Neurons

METHODS AND COMPOSITIONS FOR MODULATING GENE EXPRESSION

Self-complementary AAV vectors carrying dominant negative RhoA and...

USING MINIVECTORS TO TREAT IDIOPATHIC PULMONARY FIBROSIS

IMPORTATION OF MITOCHONDRIAL PROTEIN BY AN ENHANCED ALLOTOPIC APPROACH

FUNCTIONAL NUCLEIC ACID MOLECULE AND METHOD

COMPOSITIONS FOR AND METHODS OF IMPROVING FLUID FLUX IN THE BRAIN

TELOMERASE REVERSE TRANSCRIPTASE-BASED THERAPIES

CORONAVIRUS VACCINE

VIRAL VECTORS AND NUCLEIC ACIDS FOR REGULATED GENE THERAPY

METHODS AND COMPOSITIONS FOR TREATING TUMOR CELLS

AAV-MEDIATED EXPRESSION USING A SYNTHETIC PROMOTER AND ENHANCER

MATERIALS AND METHODS FOR TREATMENT OF PAIN RELATED DISORDERS

GLOBIN GENE THERAPY FOR TREATING HEMOGLOBINOPATHIES

Nucleic Acid Molecule Containing Incorporated Gene Encoding Fused P...

METHODS OF TREATING EYE DISEASES

METHODS AND COMPOSITIONS FOR TREATMENT OF FRIEDREICH'S ATAXIA

METHODS AND COMPOSITIONS FOR TREATING A PREMATURE STOP CODON-MEDIAT...