having a combination of backbone and sugar modifications

Industry

CPC
C12N2310/346

This industry / category may be too specific. Please go to a parent level for more data

Parent Industries

CCHEMISTRY METALLURGY C12Biochemistry C12NMICRO-ORGANISMS OR ENZYMES COMPOSITIONS THEREOF PROPAGATING, PRESERVING OR MAINTAINING MICRO-ORGANISMS MUTATION OR GENETIC ENGINEERING CULTURE MEDIA C12N2310/00Structure or type of the nucleic acid

Current Industry

C12N2310/346having a combination of backbone and sugar modifications

Industries

Patents Grantslast 30 patents

Information Patent Grant
Modulators of diacyglycerol acyltransferase 2 (DGAT2)
- Patent number 12,215,321
- Issue date Feb 4, 2025
- Ionis Pharmaceuticals, Inc.
- Eric E. Swayze
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Angelman syndrome antisense treatment
- Patent number 12,180,480
- Issue date Dec 31, 2024
- The Texas A&M University System
- Scott Victor Dindot
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Fully stabilized asymmetric siRNA
- Patent number 12,173,286
- Issue date Dec 24, 2024
- UNIVERSITY OF MASSACHUSETTS
- Anastasia Khvorova
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Modified guide RNAs
- Patent number 12,173,284
- Issue date Dec 24, 2024
- Intellia Therapeutics, Inc.
- Amy Madison Rhoden Smith
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Modulators of APOL1 expression
- Patent number 12,168,766
- Issue date Dec 17, 2024
- Astrazeneca AB
- Susan M. Freier
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Modified double-stranded RNA agents
- Patent number 12,168,767
- Issue date Dec 17, 2024
- Alnylam Pharmaceuticals, Inc.
- Martin Maier
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Antisense oligonucleotides to restore dysferlin protein expression...
- Patent number 12,163,129
- Issue date Dec 10, 2024
- University of Massachusetts
- Robert H. Brown
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Compositions and methods for modulating neuronal excitability and m...
- Patent number 12,163,130
- Issue date Dec 10, 2024
- Icahn School of Medicine at Mount Sinai
- Anne Schaefer
- A01 - AGRICULTURE FORESTRY ANIMAL HUSBANDRY HUNTING TRAPPING FISHING
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
Therapeutic compositions for treating pain via multiple targets
- Patent number 12,152,243
- Issue date Nov 26, 2024
- QUELLTX, INC.
- Graham T. Dempsey
- 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
Methods for targeting pain directed at metabolic pathways
- Patent number 12,128,062
- Issue date Oct 29, 2024
- University of Maryland, Baltimore
- Ohannes Kevork Melemedjian
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
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
Antisense oligonucleotide for targeting progranulin
- Patent number 12,104,153
- Issue date Oct 1, 2024
- HOFFMANN-LA ROCHE INC.
- Ravi Jagasia
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
PIKFYVE antisense oligonucleotides
- Patent number 12,104,159
- Issue date Oct 1, 2024
- ACURASTEM INCORPORATED
- Wen-Hsuan Chang
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Compositions for modulating tau expression
- Patent number 12,091,662
- Issue date Sep 17, 2024
- Biogen MA Inc.
- Holly Kordasiewicz
- 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
RNAi agents for inhibiting expression of PNPLA3, pharmaceutical com...
- Patent number 12,054,718
- Issue date Aug 6, 2024
- Arrowhead Pharmaceuticals, Inc.
- Anthony Nicholas
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Antisense oligonucleotides for the treatment of Stargardt disease
- Patent number 12,054,716
- Issue date Aug 6, 2024
- Stichting Katholieke Universiteit
- Robert Wilhelmus Johanna Collin
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Two-tailed self-delivering siRNA
- Patent number 12,049,627
- Issue date Jul 30, 2024
- University of Massachusetts
- Anastasia Khvorova
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Modulation of alpha synuclein expression
- Patent number 12,043,831
- Issue date Jul 23, 2024
- Ionis Pharmaceuticals, Inc.
- Susan M. Freier
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Targeted compositions
- Patent number 12,043,833
- Issue date Jul 23, 2024
- ARBUTUS BIOPHARMA CORPORATION
- James Heyes
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Compounds and methods for the modulation of proteins
- Patent number 12,030,910
- Issue date Jul 9, 2024
- Ionis Pharmaceuticals, Inc.
- Stanley T. Crooke
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Compounds and methods for modulating factor XII
- Patent number 12,018,261
- Issue date Jun 25, 2024
- Ionis Pharmaceuticals, Inc.
- Huynh-Hoa Bui
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Enhancing plasmin activity to prevent soft tissue calcification
- Patent number 12,011,455
- Issue date Jun 18, 2024
- Vanderbilt University
- Jonathan Schoenecker
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Methods and compositions for inhibiting PMP22 expression
- Patent number 11,959,080
- Issue date Apr 16, 2024
- Ionis Pharmaceuticals, Inc.
- Gene Hung
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Targeted control of pests and pathogens by plant delivery of 2′F-AN...
- Patent number 11,946,058
- Issue date Apr 2, 2024
- The United States of America, as represented by the Secretary of Agriculture
- Wayne B. Hunter
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Antisense oligonucleotides targeting SCN2A for the treatment of SCN...
- Patent number 11,939,582
- Issue date Mar 26, 2024
- RogCon, Inc.
- Steven Petrou
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
RNA interference mediated inhibition of gene expression using short...
- Patent number 11,932,854
- Issue date Mar 19, 2024
- SIRNA Therapeutics, Inc.
- Mark Cancilla
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Compounds and methods for reducing ATXN2 expression
- Patent number 11,926,825
- Issue date Mar 12, 2024
- Ionis Pharmaceuticals, Inc.
- Susan M. Freier
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE

Patents Applicationslast 30 patents

Information Patent Application
Antisense Oligonucleotide for Targeting Progranulin
- Publication number 20250051772
- Publication date Feb 13, 2025
- HOFFMANN-LA ROCHE INC.
- Ravi Jagasia
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
STEROL REGULATORY ELEMENT BINDING PROTEIN (SREBP) CHAPERONE (SCAP)...
- Publication number 20250034570
- Publication date Jan 30, 2025
- Alnylam Pharmaceuticals, Inc.
- Kevin Fitzgerald
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
MODIFIED DOUBLE-STRANDED RNA AGENTS
- Publication number 20250034572
- Publication date Jan 30, 2025
- Alnylam Pharmaceuticals, Inc.
- Martin MAIER
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
RNAi Agents for Inhibiting Expression of PNPLA3, Pharmaceutical Com...
- Publication number 20250034577
- Publication date Jan 30, 2025
- Arrowhead Pharmaceuticals, Inc.
- Anthony Nicholas
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
METHODS AND COMPOSITIONS FOR INHIBITING PMP22 EXPRESSION
- Publication number 20250027081
- Publication date Jan 23, 2025
- Ionis Pharmaceuticals, Inc.
- Gene Hung
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
RNA MODULATING OLIGONUCLEOTIDES WITH IMPROVED CHARACTERISTICS FOR T...
- Publication number 20250011784
- Publication date Jan 9, 2025
- VICO Therapeutics B.V.
- Peter Christian DE VISSER
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
MODIFIED DOUBLE-STRANDED RNA AGENTS
- Publication number 20240409942
- Publication date Dec 12, 2024
- Alnylam Pharmaceuticals, Inc.
- Martin MAIER
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
COMPOSITIONS AND METHODS FOR MODULATING APOLIPOPROTEIN C-III EXPRES...
- Publication number 20240401038
- Publication date Dec 5, 2024
- Ionis Pharmaceuticals, Inc.
- Thazha P. Prakash
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
Chemically Modified Guide RNAs for CRISPR/CAS-Mediated Gene Correction
- Publication number 20240401034
- Publication date Dec 5, 2024
- 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
Compounds and Methods for Reducing ATXN2 Expression
- Publication number 20240401039
- Publication date Dec 5, 2024
- Ionis Pharmaceuticals, Inc.
- Susan M. Freier
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
COMPLEMENT COMPONENT iRNA COMPOSITIONS AND METHODS OF USE THEREOF
- Publication number 20240376477
- Publication date Nov 14, 2024
- Alnylam Pharmaceuticals, Inc.
- Anna Borodovsky
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
GAPMER OLIGONUCLEOTIDES COMPRISING A PHOSPHORODITHIOATE INTERNUCLEO...
- Publication number 20240368593
- Publication date Nov 7, 2024
- Roche Innovation Center Copenhagen A/S
- Konrad Bleicher
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
METHODS AND MEANS FOR EFFICIENT SKIPPING OF AT LEAST ONE OF THE FOL...
- Publication number 20240360452
- Publication date Oct 31, 2024
- BioMarin Technologies B.V.
- Judith Christina Theodora Van Deutekom
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
Compounds and Methods for Reducing LRRK2 Expression
- Publication number 20240360453
- Publication date Oct 31, 2024
- Ionis Pharmaceuticals, Inc.
- Tracy A. Cole
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
LINKAGE MODIFIED OLIGOMERIC COMPOUNDS AND USES THEREOF
- Publication number 20240360448
- Publication date Oct 31, 2024
- Ionis Pharmaceuticals, Inc.
- Hans-Joachim Josef Gaus
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
COMPOSITIONS AND METHODS FOR MODULATING APOLIPOPROTEIN (a) EXPRESSION
- Publication number 20240352455
- Publication date Oct 24, 2024
- Ionis Pharmaceuticals, Inc.
- Thazha P. Prakash
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
MODULATORS OF ENaC EXPRESSION
- Publication number 20240327838
- Publication date Oct 3, 2024
- Ionis Pharmaceuticals, Inc.
- Jeffrey R. Crosby
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
CRISPR-BASED COMPOSITIONS AND METHODS OF USE
- Publication number 20240318171
- Publication date Sep 26, 2024
- Integrated DNA Technologies, Inc.
- Michael Allen Collingwood
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
COMPOUNDS AND METHODS FOR INCREASING STMN2 EXPRESSION
- Publication number 20240316092
- Publication date Sep 26, 2024
- Ionis Pharmaceuticals, Inc.
- Huynh-Hoa Bui
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
COMPOSITIONS FOR MODULATING ATAXIN 2 EXPRESSION
- Publication number 20240309374
- Publication date Sep 19, 2024
- Ionis Pharmaceuticals, Inc.
- Susan M. Freier
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF TMPRSS6 GENE
- Publication number 20240309386
- Publication date Sep 19, 2024
- Alnylam Pharmaceuticals, Inc.
- David Bumcrot
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
REDUCING INTRON RETENTION
- Publication number 20240309377
- Publication date Sep 19, 2024
- UNIVERSITY OF SOUTHAMPTON
- Igor Vorechovsky
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
COMPLEMENT COMPONENT C5 iRNA COMPOSITIONS AND METHODS OF USE THEREO...
- Publication number 20240301406
- Publication date Sep 12, 2024
- Alnylam Pharmaceuticals, Inc.
- Nader Najafian
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
COMPOUNDS AND METHODS FOR REDUCING ATXN3 EXPRESSION
- Publication number 20240301412
- Publication date Sep 12, 2024
- Ionis Pharmaceuticals, Inc.
- Susan M. Freier
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
COMPOSITIONS AND METHODS FOR CORRECTING LIMB GIRDLE MUSCULAR DYSTRO...
- Publication number 20240301428
- Publication date Sep 12, 2024
- The University of Chicago
- Elizabeth McNally
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
ANTISENSE OLIGONUCLEOTIDES TARGETING SCN2A FOR THE TREATMENT OF SCN...
- Publication number 20240294922
- Publication date Sep 5, 2024
- RogCon, Inc.
- Steven Petrou
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING SHORT...
- Publication number 20240287515
- Publication date Aug 29, 2024
- Srina Therapeutics, Inc.
- Mark Cancilla
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
Compounds and Methods for Modulating Splicing of Pre-MRNA
- Publication number 20240287513
- Publication date Aug 29, 2024
- Ionis Pharmaceuticals, Inc.
- Frank Rigo
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
RNAi AGENTS, COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING T...
- Publication number 20240279652
- Publication date Aug 22, 2024
- Alnylam Pharmaceuticals, Inc.
- Kallanthottathil G. Rajeev
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
METHODS FOR THE TREATMENT OF NUCLEOTIDE REPEAT EXPANSION DISORDERS...
- Publication number 20240263179
- Publication date Aug 8, 2024
- Takeda Pharmaceuticals U.S.A., Inc
- Nessan Anthony BERMINGHAM
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...

having a combination of backbone and sugar modifications

Industry

Parent Industries

Current Industry

Patents Grantslast 30 patents

Modulators of diacyglycerol acyltransferase 2 (DGAT2)

Angelman syndrome antisense treatment

Fully stabilized asymmetric siRNA

Modified guide RNAs

Modulators of APOL1 expression

Modified double-stranded RNA agents

Antisense oligonucleotides to restore dysferlin protein expression...

Compositions and methods for modulating neuronal excitability and m...

Selective antisense compounds and uses thereof

Therapeutic compositions for treating pain via multiple targets

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

Methods for targeting pain directed at metabolic pathways

Selective reduction of allelic variants

Antisense oligonucleotide for targeting progranulin

PIKFYVE antisense oligonucleotides

Compositions for modulating tau expression

SERPINA1 iRNA compositions and methods of use thereof

RNAi agents for inhibiting expression of PNPLA3, pharmaceutical com...

Antisense oligonucleotides for the treatment of Stargardt disease

Two-tailed self-delivering siRNA

Modulation of alpha synuclein expression

Targeted compositions

Compounds and methods for the modulation of proteins

Compounds and methods for modulating factor XII

Enhancing plasmin activity to prevent soft tissue calcification

Methods and compositions for inhibiting PMP22 expression

Targeted control of pests and pathogens by plant delivery of 2′F-AN...

Antisense oligonucleotides targeting SCN2A for the treatment of SCN...

RNA interference mediated inhibition of gene expression using short...

Compounds and methods for reducing ATXN2 expression

Patents Applicationslast 30 patents

Antisense Oligonucleotide for Targeting Progranulin

STEROL REGULATORY ELEMENT BINDING PROTEIN (SREBP) CHAPERONE (SCAP)...

MODIFIED DOUBLE-STRANDED RNA AGENTS

RNAi Agents for Inhibiting Expression of PNPLA3, Pharmaceutical Com...

METHODS AND COMPOSITIONS FOR INHIBITING PMP22 EXPRESSION

RNA MODULATING OLIGONUCLEOTIDES WITH IMPROVED CHARACTERISTICS FOR T...

MODIFIED DOUBLE-STRANDED RNA AGENTS

COMPOSITIONS AND METHODS FOR MODULATING APOLIPOPROTEIN C-III EXPRES...

Chemically Modified Guide RNAs for CRISPR/CAS-Mediated Gene Correction

Compounds and Methods for Reducing ATXN2 Expression

COMPLEMENT COMPONENT iRNA COMPOSITIONS AND METHODS OF USE THEREOF

GAPMER OLIGONUCLEOTIDES COMPRISING A PHOSPHORODITHIOATE INTERNUCLEO...

METHODS AND MEANS FOR EFFICIENT SKIPPING OF AT LEAST ONE OF THE FOL...

Compounds and Methods for Reducing LRRK2 Expression

LINKAGE MODIFIED OLIGOMERIC COMPOUNDS AND USES THEREOF

COMPOSITIONS AND METHODS FOR MODULATING APOLIPOPROTEIN (a) EXPRESSION

MODULATORS OF ENaC EXPRESSION

CRISPR-BASED COMPOSITIONS AND METHODS OF USE

COMPOUNDS AND METHODS FOR INCREASING STMN2 EXPRESSION

COMPOSITIONS FOR MODULATING ATAXIN 2 EXPRESSION

COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF TMPRSS6 GENE

REDUCING INTRON RETENTION

COMPLEMENT COMPONENT C5 iRNA COMPOSITIONS AND METHODS OF USE THEREO...

COMPOUNDS AND METHODS FOR REDUCING ATXN3 EXPRESSION

COMPOSITIONS AND METHODS FOR CORRECTING LIMB GIRDLE MUSCULAR DYSTRO...

ANTISENSE OLIGONUCLEOTIDES TARGETING SCN2A FOR THE TREATMENT OF SCN...

RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING SHORT...

Compounds and Methods for Modulating Splicing of Pre-MRNA

RNAi AGENTS, COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING T...

METHODS FOR THE TREATMENT OF NUCLEOTIDE REPEAT EXPANSION DISORDERS...