Allele or polymorphism specific uses

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C12N2320/34

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CCHEMISTRY METALLURGY C12Biochemistry C12NMICRO-ORGANISMS OR ENZYMES COMPOSITIONS THEREOF PROPAGATING, PRESERVING OR MAINTAINING MICRO-ORGANISMS MUTATION OR GENETIC ENGINEERING CULTURE MEDIA C12N2320/00Applications Uses

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C12N2320/34Allele or polymorphism specific uses

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Patents Grantslast 30 patents

Information Patent Grant
Selective antisense compounds and uses thereof
- Patent number 12,152,244
- Issue date Nov 26, 2024
- Ionis Pharmaceuticals, Inc.
- Michael Oestergaard
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Antisense oligonucleotides for the treatment of Leber congenital am...
- Patent number 12,139,711
- Issue date Nov 12, 2024
- STICHTING RADBOUD UNIVERSITAIR MEDISCH CENTRUM
- Robert Wilhelmus Johanna Collin
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Selective reduction of allelic variants
- Patent number 12,110,491
- Issue date Oct 8, 2024
- Ionis Pharmaceuticals, Inc.
- C. Frank Bennett
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
SERPINA1 iRNA compositions and methods of use thereof
- Patent number 12,071,619
- Issue date Aug 27, 2024
- Alnylam Pharmaceuticals, Inc.
- Alfica Sehgal
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Methods and compositions for reducing pathogenic isoforms
- Patent number 12,043,832
- Issue date Jul 23, 2024
- Washington University
- Andrew Findlay
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Trans-splicing molecules
- Patent number 11,993,776
- Issue date May 28, 2024
- Ascidian Therapeutics, Inc.
- Philip R. Johnson
- C07 - ORGANIC CHEMISTRY
Information Patent Grant
RNA targeting of mutations via suppressor tRNAs and deaminases
- Patent number 11,932,856
- Issue date Mar 19, 2024
- The Regents of the University of California
- Prashant Mali
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Allele-specific silencing therapy for dynamin 2-related diseases
- Patent number 11,926,829
- Issue date Mar 12, 2024
- ASSOICATION INSTITUT DE MYOLOGIE
- Marc Bitoun
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Methods for reactivating genes on the inactive X chromosome
- Patent number 11,912,994
- Issue date Feb 27, 2024
- The General Hospital Corporation
- Jeannie T. Lee
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Antisense oligonucleotides useful in treatment of Pompe disease
- Patent number 11,859,186
- Issue date Jan 2, 2024
- Erasmus University Medical Center Rotterdam
- Atze Jacobus Bergsma
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Oligonucleotides for inducing paternal UBE3A expression
- Patent number 11,852,627
- Issue date Dec 26, 2023
- HOFFMANN-LA ROCHE INC.
- Veronica Costa
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Chemically modified single-stranded RNA-editing oligonucleotides
- Patent number 11,851,656
- Issue date Dec 26, 2023
- ProQR Therapeutics II B.V.
- Janne Juha Turunen
- 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
Allele selective inhibition of mutant C9orf72 foci expression by du...
- Patent number 11,840,690
- Issue date Dec 12, 2023
- The Board of Regents of the University of Texas System
- David Corey
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Repairing compound heterozygous recessive mutations by allele exchange
- Patent number 11,773,410
- Issue date Oct 3, 2023
- University of Massachusetts
- Guangping Gao
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
HSD17B13 variants and uses thereof
- Patent number 11,753,628
- Issue date Sep 12, 2023
- REGENERON PHARMACEUTICALS, INC.
- Noura S. Abul-Husn
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Allele specific modulators of P23H rhodopsin
- Patent number 11,744,846
- Issue date Sep 5, 2023
- Susan F. Murray
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Selective antisense compounds and uses thereof
- Patent number 11,732,261
- Issue date Aug 22, 2023
- Ionis Pharmaceuticals, Inc.
- Punit P. Seth
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Compositions and methods for increasing expression of SCN2A
- Patent number 11,713,463
- Issue date Aug 1, 2023
- The Florey Institute of Neuroscience and Mental Health
- Steven Petrou
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Modulation of gene expression and screening for deregulated protein...
- Patent number 11,702,660
- Issue date Jul 18, 2023
- University of Southampton
- Igor Vorechovsky
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Oligonucleotides for treating expanded repeat diseases
- Patent number 11,674,139
- Issue date Jun 13, 2023
- Sarepta Therapeutics, Inc.
- Peter Linsley
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Gene therapeutics for fibrodysplasia ossificans progressiva
- Patent number 11,674,154
- Issue date Jun 13, 2023
- University of Massachusetts
- Jae-Hyuck Shim
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
RNA targeting of mutations via suppressor tRNAs and deaminases
- Patent number 11,608,503
- Issue date Mar 21, 2023
- The Regents of the University of California
- Prashant Mali
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Method of allele specific silencing for the treatment of autosomal...
- Patent number 11,591,602
- Issue date Feb 28, 2023
- Istituti Clinici Scientifici Maugeri SpA SB
- Silvia G. Priori
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Application of microRNA-210 inhibitor in preparation of drugs for t...
- Patent number 11,542,502
- Issue date Jan 3, 2023
- THE SECOND XIANGYA HOSPITAL OF CENTRAL SOUTH UNIVERSITY
- Qianjin Lu
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Methods for improving the health of porcine species by targeted ina...
- Patent number 11,535,850
- Issue date Dec 27, 2022
- GENUS PLC
- Andrew Mark Cigan
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Methods for the treatment of Leber congenital amaurosis
- Patent number 11,492,621
- Issue date Nov 8, 2022
- Institut National de la Sante et de la Recherche Medicale
- Jean-Michel Rozet
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Compositions and methods for targeting cancer-specific sequence var...
- Patent number 11,492,670
- Issue date Nov 8, 2022
- The Broad Institute Inc.
- William C. Hahn
- B82 - NANO-TECHNOLOGY
Information Patent Grant
HSD17B13 variants and uses thereof
- Patent number 11,485,958
- Issue date Nov 1, 2022
- REGENERON PHARMACEUTICALS, INC.
- Noura S. Abul-Husn
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
RNA targeting of mutations via suppressor tRNAs and deaminases
- Patent number 11,479,775
- Issue date Oct 25, 2022
- The Regents of the University of California
- Prashant Mali
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...

Patents Applicationslast 30 patents

Information Patent Application
RNAi TARGETING KIF1A MISSENSE MUTATIONS FOR TREATMENT OF KIF1A ASSO...
- Publication number 20240409936
- Publication date Dec 12, 2024
- Ovid Therapeutics Inc.
- Noelle Germain
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
CORRECTION OF SPLICING MUTATIONS CAUSING PRIMARY CILIARY DYSKINESIA...
- Publication number 20240409932
- Publication date Dec 12, 2024
- The University of North Carolina at Chapel Hill
- Silvia Kreda
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
RNA TARGETING OF MUTATIONS VIA SUPPRESSOR tRNAs AND DEAMINASES
- Publication number 20240392303
- Publication date Nov 28, 2024
- The Regents of the University of California
- Prashant Mali
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
Selective Antisense Compounds and Uses Thereof
- Publication number 20240368592
- Publication date Nov 7, 2024
- Ionis Pharmaceuticals, Inc.
- Punit P. Seth
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
RNA TARGETING OF MUTATIONS VIA SUPPRESSOR tRNAs AND DEAMINASES
- Publication number 20240352465
- Publication date Oct 24, 2024
- The Regents of the University of California
- Prashant Mali
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
METHODS FOR REACTIVATING GENES ON THE INACTIVE X CHROMOSOME
- Publication number 20240327835
- Publication date Oct 3, 2024
- The General Hospital Corporation
- Jeannie T. Lee
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
TRANS-SPLICING MOLECULES
- Publication number 20240318186
- Publication date Sep 26, 2024
- Ascidian Therapeutics, Inc.
- Philip R. JOHNSON
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
Selective Reduction of Allelic Variants
- Publication number 20240309452
- Publication date Sep 19, 2024
- Ionis Pharmaceuticals, Inc.
- C. Frank Bennett
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
Selective Antisense Compounds and Uses Thereof
- Publication number 20240294923
- Publication date Sep 5, 2024
- Ionis Pharmaceuticals, Inc.
- Michael Oestergaard
- C07 - ORGANIC CHEMISTRY
Information Patent Application
Allele-specific silencing therapy for DFNA21 using antisense oligon...
- Publication number 20240287521
- Publication date Aug 29, 2024
- Stichting Radboud universitair medisch centrum
- Hendrikus Antonius Rudolfus Van Wyk
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
CHEMICALLY MODIFIED SINGLE-STRANDED RNA-EDITING OLIGONUCLEOTIDES
- Publication number 20240271131
- Publication date Aug 15, 2024
- ProQR Therapeutics II B.V.
- Janne Juha Turunen
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
Methods and Compositions for Adar-Mediated Editing
- Publication number 20240263177
- Publication date Aug 8, 2024
- Korro Bio, Inc.
- Andrew Fraley
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
ALLELE-SPECIFIC SILENCING THERAPY FOR DYNAMIN 2-RELATED DISEASES
- Publication number 20240229039
- Publication date Jul 11, 2024
- ASSOCIATION INSTITUT DE MYOLOGIE
- Marc BITOUN
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
OLIGONUCLEOTIDES FOR TREATING EXPANDED REPEAT DISEASES
- Publication number 20240209363
- Publication date Jun 27, 2024
- Sarepta Therapeutics, Inc.
- Peter Linsley
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
TRANSTHYRETIN (TTR) iRNA COMPOSITIONS AND METHODS OF USE THEREOF FO...
- Publication number 20240200061
- Publication date Jun 20, 2024
- Alnylam Pharmaceuticals, Inc.
- Jayaprakash K. Nair
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
Compositions and Methods for Treatment of Myotonic Dystrophy Type 1...
- Publication number 20240181081
- Publication date Jun 6, 2024
- VERTEX PHARMACEUTICALS INCORPORATED
- Guoxiang Ruan
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
COMPLEMENT COMPONENT C5 IRNA COMPOSITIONS AND METHODS OF USE THEREOF
- Publication number 20240175017
- Publication date May 30, 2024
- Alnylam Pharmaceuticals, Inc.
- Anna Borodovsky
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
COMPOSITIONS OF MODIFIED TREMS AND USES THEREOF
- Publication number 20240175020
- Publication date May 30, 2024
- Flagship Pioneering Innovations VI, LLC.
- Theonie Anastassiadis
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
METHOD FOR GENE REPAIR IN PRIMARY HUMAN MUSCLE STEM CELLS (SATELLIT...
- Publication number 20240150718
- Publication date May 9, 2024
- Charite-Universitaetsmedizin Berlin
- Simone SPULER
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
HSD17B13 VARIANTS AND USES THEREOF
- Publication number 20240141305
- Publication date May 2, 2024
- Regeneron Pharmaceuticals, Inc.
- Noura S. Abul-Husn
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
Selective Antisense Compounds and Uses Thereof
- Publication number 20240141343
- Publication date May 2, 2024
- Ionis Pharmaceuticals, Inc.
- Punit P. Seth
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
ALLELE SELECTIVE INHIBITION OF MUTANT C9ORF72 FOCI EXPRESSION BY DU...
- Publication number 20240141348
- Publication date May 2, 2024
- The Board of Regents of the University of Texas System
- David COREY
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
COMPOUND AND METHOD FOR AN ALLELE-SPECIFIC EDITING OF THE ELANE GENE
- Publication number 20240139342
- Publication date May 2, 2024
- Eberhard Karls Universität Tübingen Medizinische Fakultät
- Julia Skokowa
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
COMPOSITIONS AND METHODS FOR TREATING TRANSTHYRETIN AMYLOIDOSIS
- Publication number 20240117345
- Publication date Apr 11, 2024
- Beam Therapeutics Inc.
- Michael PACKER
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
Suppression-Replacement Gene Therapy
- Publication number 20240093202
- Publication date Mar 21, 2024
- Mayo Foundation for Medical Education and Research
- Michael J. ACKERMAN
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
OLIGONUCLEOTIDES FOR INDUCING PATERNAL UBE3A EXPRESSION
- Publication number 20240085402
- Publication date Mar 14, 2024
- HOFFMANN-LA ROCHE INC.
- Veronica COSTA
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
METHOD OF ALLELE SPECIFIC SILENCING FOR THE TREATMENT OF AUTOSOMAL...
- Publication number 20240067974
- Publication date Feb 29, 2024
- Istituti Clinici Scientifici Maugeri SpA SB
- Silvia G. PRIORI
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
COMPOSITIONS AND METHODS FOR SILENCING KRAS
- Publication number 20240060069
- Publication date Feb 22, 2024
- Nanothera Biosciences, Inc.
- Raj CHAKRABARTI
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
ALLELE-SPECIFIC GENOME EDITING OF THE NR2E3 MUTATION G56R
- Publication number 20240052372
- Publication date Feb 15, 2024
- UNIVERSITE DE MONTPELLIER
- Vasiliki KALATZIS
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
OLIGONUCLEOTIDE COMPOSITIONS AND METHODS THEREOF
- Publication number 20240026358
- Publication date Jan 25, 2024
- WAVE LIFE SCIENCES LTD.
- Prashant Monian
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...

Allele or polymorphism specific uses

Industry

Parent Industries

Current Industry

Patents Grantslast 30 patents

Selective antisense compounds and uses thereof

Antisense oligonucleotides for the treatment of Leber congenital am...

Selective reduction of allelic variants

SERPINA1 iRNA compositions and methods of use thereof

Methods and compositions for reducing pathogenic isoforms

Trans-splicing molecules

RNA targeting of mutations via suppressor tRNAs and deaminases

Allele-specific silencing therapy for dynamin 2-related diseases

Methods for reactivating genes on the inactive X chromosome

Antisense oligonucleotides useful in treatment of Pompe disease

Oligonucleotides for inducing paternal UBE3A expression

Chemically modified single-stranded RNA-editing oligonucleotides

HSD17B13 variants and uses thereof

Allele selective inhibition of mutant C9orf72 foci expression by du...

Repairing compound heterozygous recessive mutations by allele exchange

HSD17B13 variants and uses thereof

Allele specific modulators of P23H rhodopsin

Selective antisense compounds and uses thereof

Compositions and methods for increasing expression of SCN2A

Modulation of gene expression and screening for deregulated protein...

Oligonucleotides for treating expanded repeat diseases

Gene therapeutics for fibrodysplasia ossificans progressiva

RNA targeting of mutations via suppressor tRNAs and deaminases

Method of allele specific silencing for the treatment of autosomal...

Application of microRNA-210 inhibitor in preparation of drugs for t...

Methods for improving the health of porcine species by targeted ina...

Methods for the treatment of Leber congenital amaurosis

Compositions and methods for targeting cancer-specific sequence var...

HSD17B13 variants and uses thereof

RNA targeting of mutations via suppressor tRNAs and deaminases

Patents Applicationslast 30 patents

RNAi TARGETING KIF1A MISSENSE MUTATIONS FOR TREATMENT OF KIF1A ASSO...

CORRECTION OF SPLICING MUTATIONS CAUSING PRIMARY CILIARY DYSKINESIA...

RNA TARGETING OF MUTATIONS VIA SUPPRESSOR tRNAs AND DEAMINASES

Selective Antisense Compounds and Uses Thereof

RNA TARGETING OF MUTATIONS VIA SUPPRESSOR tRNAs AND DEAMINASES

METHODS FOR REACTIVATING GENES ON THE INACTIVE X CHROMOSOME

TRANS-SPLICING MOLECULES

Selective Reduction of Allelic Variants

Selective Antisense Compounds and Uses Thereof

Allele-specific silencing therapy for DFNA21 using antisense oligon...

CHEMICALLY MODIFIED SINGLE-STRANDED RNA-EDITING OLIGONUCLEOTIDES

Methods and Compositions for Adar-Mediated Editing

ALLELE-SPECIFIC SILENCING THERAPY FOR DYNAMIN 2-RELATED DISEASES

OLIGONUCLEOTIDES FOR TREATING EXPANDED REPEAT DISEASES

TRANSTHYRETIN (TTR) iRNA COMPOSITIONS AND METHODS OF USE THEREOF FO...

Compositions and Methods for Treatment of Myotonic Dystrophy Type 1...

COMPLEMENT COMPONENT C5 IRNA COMPOSITIONS AND METHODS OF USE THEREOF

COMPOSITIONS OF MODIFIED TREMS AND USES THEREOF

METHOD FOR GENE REPAIR IN PRIMARY HUMAN MUSCLE STEM CELLS (SATELLIT...

HSD17B13 VARIANTS AND USES THEREOF

Selective Antisense Compounds and Uses Thereof

ALLELE SELECTIVE INHIBITION OF MUTANT C9ORF72 FOCI EXPRESSION BY DU...

COMPOUND AND METHOD FOR AN ALLELE-SPECIFIC EDITING OF THE ELANE GENE

COMPOSITIONS AND METHODS FOR TREATING TRANSTHYRETIN AMYLOIDOSIS

Suppression-Replacement Gene Therapy

OLIGONUCLEOTIDES FOR INDUCING PATERNAL UBE3A EXPRESSION

METHOD OF ALLELE SPECIFIC SILENCING FOR THE TREATMENT OF AUTOSOMAL...

COMPOSITIONS AND METHODS FOR SILENCING KRAS

ALLELE-SPECIFIC GENOME EDITING OF THE NR2E3 MUTATION G56R

OLIGONUCLEOTIDE COMPOSITIONS AND METHODS THEREOF