Alteration of splicing

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C12N2320/33

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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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C12N2320/33Alteration of splicing

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Patents Grantslast 30 patents

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
Ventricular arrhythmias and related methods
- Patent number 12,207,932
- Issue date Jan 28, 2025
- UNIVERSITY OF MASSACHUSETTS
- John Kevin Donahue
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Antisense oligonucleotides for modulating the function of a T cell
- Patent number 12,188,016
- Issue date Jan 7, 2025
- Agency for Science, Technology and Research
- Ernest Guccione
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Antisense oligonucleotides for the treatment of Stargardt disease
- Patent number 12,180,471
- Issue date Dec 31, 2024
- ProQR Therapeutics II B.V.
- Aliye Seda Yilmaz-Elis
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Compositions and uses for treatment thereof
- Patent number 12,163,128
- Issue date Dec 10, 2024
- Board of Supervisors of Louisiana State University and Agricultural and Mecha...
- Edward L. Grabczyk
- 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
RNA editing via recruitment of spliceosome components
- Patent number 12,152,239
- Issue date Nov 26, 2024
- Tacit Therapeutics, Inc.
- David A. Nelles
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Direct and selective inhibition of MDM4 for treatment of cancer
- Patent number 12,139,712
- Issue date Nov 12, 2024
- VIB VZW
- Jean-Christophe Marine
- 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
Antisense oligomers and methods of using the same for treating dise...
- Patent number 12,139,709
- Issue date Nov 12, 2024
- Sarepta Therapeutics, Inc.
- Frederick Joseph Schnell
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Nucleic acids and methods for the treatment of Pompe disease
- Patent number 12,091,665
- Issue date Sep 17, 2024
- SYNTHENA AG
- Luis Garcia
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Methods for using transcription-dependent directed evolution of AAV...
- Patent number 12,084,654
- Issue date Sep 10, 2024
- Oregon Health & Science University
- Hiroyuki Nakai
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Methods for using transcription-dependent directed evolution of AAV...
- Patent number 12,084,653
- Issue date Sep 10, 2024
- Oregon Health & Science University
- Hiroyuki Nakai
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Recombinant virus products and methods for inducing DUX4 exon skipping
- Patent number 12,084,658
- Issue date Sep 10, 2024
- Research Institute at Nationwide Children's Hospital
- Scott Quenton Harper
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Anti-transferrin receptor antibody-PMO conjugates for inducing DMD...
- Patent number 12,071,621
- Issue date Aug 27, 2024
- AVIDITY BIOSCIENCES, INC.
- Beatrice Diana Darimont
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Splice-switching oligonucleotides and methods of use
- Patent number 12,071,626
- Issue date Aug 27, 2024
- Duke University
- Jennifer Freedman
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Nucleic acid-polypeptide compositions and methods of inducing exon...
- Patent number 12,064,483
- Issue date Aug 20, 2024
- AVIDITY BIOSCIENCES, INC.
- Arthur A. Levin
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Antisense nucleic acids
- Patent number 12,060,556
- Issue date Aug 13, 2024
- Nippon Shinyaku Co., Ltd.
- Naoki Watanabe
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Methods and compositions for treatment of cholesteryl ester storage...
- Patent number 12,060,558
- Issue date Aug 13, 2024
- Stoke Therapeutics, Inc.
- Isabel Aznarez
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
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
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
Antisense compounds targeting genes associated with cystic fibrosis
- Patent number 12,042,508
- Issue date Jul 23, 2024
- Rosalind Franklin University of Medicine and Science
- Michelle L. Hastings
- A01 - AGRICULTURE FORESTRY ANIMAL HUSBANDRY HUNTING TRAPPING FISHING
Information Patent Grant
Systems, methods, and compositions for RNA-guided RNA-targeting CRI...
- Patent number 12,012,621
- Issue date Jun 18, 2024
- Massachusetts Institute of Technology
- Omar Abudayyeh
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
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
Methods of synthesizing oligonucleotides using tethered nucleotides
- Patent number 11,970,697
- Issue date Apr 30, 2024
- Twist Bioscience Corporation
- Jeremy Lackey
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Antisense oligonucleotide and composition for prevention or treatme...
- Patent number 11,963,974
- Issue date Apr 23, 2024
- National Center for Child Health and Development
- Go Tajima
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Polynucleotide compositions and methods for gene expression regulat...
- Patent number 11,946,050
- Issue date Apr 2, 2024
- APTAH BIO, INC.
- Caio Bruno Quinta De Souza Leal
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Exon skipping compositions for treating muscular dystrophy
- Patent number 11,932,851
- Issue date Mar 19, 2024
- Sarepta Therapeutics, Inc.
- Richard K. Bestwick
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Oligonucleotide therapy for Leber congenital amaurosis
- Patent number 11,920,132
- Issue date Mar 5, 2024
- ProQR Therapeutics II B.V.
- Patricia Coromoto Biasutto
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Bicyclic nucleosides and oligomers prepared therefrom
- Patent number 11,919,922
- Issue date Mar 5, 2024
- Universitat Bern
- Christian Leumann
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...

Patents Applicationslast 30 patents

Information Patent Application
ANTISENSE NUCLEIC ACIDS
- Publication number 20250042933
- Publication date Feb 6, 2025
- NIPPON SHINYAKU CO., LTD
- Naoki Watanabe
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
ANTISENSE OLIGONUCLEOTIDE FOR REGULATING BCL2L12 EXPRESSION AND THE...
- Publication number 20250019707
- Publication date Jan 16, 2025
- SHANDONG UNIVERSITY
- Zhaojian LIU
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
NEURODEGENERATIVE DISORDERS
- Publication number 20250011772
- Publication date Jan 9, 2025
- Teesside University
- Pavlos Alifragis
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
COMPOSITIONS AND METHODS FOR SKIPPING EXON 45 IN DUCHENNE MUSCULAR...
- Publication number 20240417429
- Publication date Dec 19, 2024
- Entrada Therapeutics, Inc.
- Xiang Li
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
ANTISENSE-INDUCED EXON EXCLUSION IN TYPE VII COLLAGEN
- Publication number 20240415857
- Publication date Dec 19, 2024
- Sarepta Therapeutics, Inc.
- Dan V. Mourich
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
NOVEL RNA TRANSCRIPT
- Publication number 20240409928
- Publication date Dec 12, 2024
- PTC THERAPEUTICS, INC.
- Anuradha BHATTACHARYYA
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
RNA-GUIDED TRANS-SPLICING OF RNA
- Publication number 20240409920
- Publication date Dec 12, 2024
- THE BROAD INSTITUTE, INC.
- Feng Zhang
- C07 - ORGANIC CHEMISTRY
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
THERAPEUTICS FOR THE TREATMENT OF NEURODEGENERATIVE DISORDERS
- Publication number 20240401036
- Publication date Dec 5, 2024
- UCL Business LTD
- Pietro Fratta
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
AGENTS USEFUL IN TREATING FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY
- Publication number 20240401037
- Publication date Dec 5, 2024
- Universite de Mons
- Alexandra Belayew
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
NUCLEOTIDE AND USE THEREOF
- Publication number 20240392291
- Publication date Nov 28, 2024
- GUANGZHOU REFORGENE MEDICINE CO., LTD.
- Junbin Liang
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
Nucleic Acids and Methods for the Treatment of Pompe Disease
- Publication number 20240392300
- Publication date Nov 28, 2024
- SYNTHENA AG
- Luis Garcia
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
TARGETED GENE THERAPY FOR DM-1 MYOTONIC DYSTROPHY
- Publication number 20240384271
- Publication date Nov 21, 2024
- Genzyme Corporation
- Seng Cheng
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
CONJUGATED OLIGONUCLEOTIDES AND USES THEREOF
- Publication number 20240382605
- Publication date Nov 21, 2024
- Ionis Pharmaceuticals, Inc.
- Punit P. Seth
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
MUSCLE TARGETING COMPLEXES AND USES THEREOF FOR TREATING DYSTROPHIN...
- Publication number 20240382609
- Publication date Nov 21, 2024
- Dyne Therapeutics, Inc.
- Cody A. Desjardins
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
NUCLEIC ACID MOLECULE AND ITS USE
- Publication number 20240384270
- Publication date Nov 21, 2024
- RNA MORPH LTD
- Eitan LEV
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
ANTI-TRANSFERRIN RECEPTOR ANTIBODY-PMO CONJUGATES FOR INDUCING DMD...
- Publication number 20240368598
- Publication date Nov 7, 2024
- Avidity Biosciences, Inc.
- Beatrice Diana DARIMONT
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
USE OF SPLICE SWITCHING OLIGONUCLEOTIDES FOR EXON SKIPPING-MEDIATED...
- Publication number 20240368605
- Publication date Nov 7, 2024
- INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE
- Thierry FEST
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
MUSCLE TARGETING COMPLEXES AND USES THEREOF FOR TREATING DYSTROPHIN...
- Publication number 20240368296
- Publication date Nov 7, 2024
- Dyne Therapeutics, Inc.
- Cody A. Desjardins
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
OLIGOMERIC COMPOUND FOR DYSTROPHIN RESCUE IN DMD PATIENTS THROUGHOU...
- Publication number 20240368591
- Publication date Nov 7, 2024
- SQY THERAPEUTICS
- Luis GARCIA
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
COMPOUNDS AND METHODS FOR MODULATING SCN1A EXPRESSION
- Publication number 20240360449
- Publication date Oct 31, 2024
- Ionis Pharmaceuticals, Inc.
- Paymaan Jafar-nejad
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
ANTISENSE OLIGONUCLEOTIDE THERAPY FOR H3.3 K27M DIFFUSE MIDLINE GLI...
- Publication number 20240360450
- Publication date Oct 31, 2024
- COLD SPRING HARBOR HARBOR LABORATORY
- Adrian Krainer
- 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
NUCLEIC ACID-POLYPEPTIDE COMPOSITIONS AND METHODS OF INDUCING EXON...
- Publication number 20240358736
- Publication date Oct 31, 2024
- Avidity Biosciences, Inc.
- Arthur A. LEVIN
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
SNRNA TARGETING USH2A PRE-MRNA AND APPLICATION THEREOF
- Publication number 20240352460
- Publication date Oct 24, 2024
- GUANGZHOU REFORGENE MEDICINE CO., LTD.
- Junbin Liang
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
METHODS OF SYNTHESIZING OLIGONUCLEOTIDES USING TETHERED NUCLEOTIDES
- Publication number 20240344057
- Publication date Oct 17, 2024
- Twist Bioscience Corporation
- Jeremy LACKEY
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
NOVEL BICYCLIC NUCLEOSIDES AND OLIGOMERS PREPARED THEREFROM
- Publication number 20240343752
- Publication date Oct 17, 2024
- Universitat Bern
- Christian Leumann
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
METHOD
- Publication number 20240344065
- Publication date Oct 17, 2024
- Oxford University Innovation Limited
- Charles Thomas ROBERTS
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
COMPOSITIONS COMPRISING EXON SKIPPING OLIGONUCLEOTIDE CONJUGATES FO...
- Publication number 20240327831
- Publication date Oct 3, 2024
- Sarepta Therapeutics, Inc.
- Arani CHANDA
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
ANTISENSE POLYNUCLEOTIDES TO INDUCE EXON SKIPPING AND METHOD OF TRE...
- Publication number 20240327844
- Publication date Oct 3, 2024
- The University of Chicago
- Elizabeth McNally
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...

Alteration of splicing

Industry

Parent Industries

Current Industry

Patents Grantslast 30 patents

Therapeutic applications of CPF1-based genome editing

Ventricular arrhythmias and related methods

Antisense oligonucleotides for modulating the function of a T cell

Antisense oligonucleotides for the treatment of Stargardt disease

Compositions and uses for treatment thereof

Antisense oligonucleotides to restore dysferlin protein expression...

RNA editing via recruitment of spliceosome components

Direct and selective inhibition of MDM4 for treatment of cancer

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

Antisense oligomers and methods of using the same for treating dise...

Nucleic acids and methods for the treatment of Pompe disease

Methods for using transcription-dependent directed evolution of AAV...

Methods for using transcription-dependent directed evolution of AAV...

Recombinant virus products and methods for inducing DUX4 exon skipping

Anti-transferrin receptor antibody-PMO conjugates for inducing DMD...

Splice-switching oligonucleotides and methods of use

Nucleic acid-polypeptide compositions and methods of inducing exon...

Antisense nucleic acids

Methods and compositions for treatment of cholesteryl ester storage...

Antisense oligonucleotides for the treatment of Stargardt disease

Methods and compositions for reducing pathogenic isoforms

Antisense compounds targeting genes associated with cystic fibrosis

Systems, methods, and compositions for RNA-guided RNA-targeting CRI...

Trans-splicing molecules

Methods of synthesizing oligonucleotides using tethered nucleotides

Antisense oligonucleotide and composition for prevention or treatme...

Polynucleotide compositions and methods for gene expression regulat...

Exon skipping compositions for treating muscular dystrophy

Oligonucleotide therapy for Leber congenital amaurosis

Bicyclic nucleosides and oligomers prepared therefrom

Patents Applicationslast 30 patents

ANTISENSE NUCLEIC ACIDS

ANTISENSE OLIGONUCLEOTIDE FOR REGULATING BCL2L12 EXPRESSION AND THE...

NEURODEGENERATIVE DISORDERS

COMPOSITIONS AND METHODS FOR SKIPPING EXON 45 IN DUCHENNE MUSCULAR...

ANTISENSE-INDUCED EXON EXCLUSION IN TYPE VII COLLAGEN

NOVEL RNA TRANSCRIPT

RNA-GUIDED TRANS-SPLICING OF RNA

CORRECTION OF SPLICING MUTATIONS CAUSING PRIMARY CILIARY DYSKINESIA...

THERAPEUTICS FOR THE TREATMENT OF NEURODEGENERATIVE DISORDERS

AGENTS USEFUL IN TREATING FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY

NUCLEOTIDE AND USE THEREOF

Nucleic Acids and Methods for the Treatment of Pompe Disease

TARGETED GENE THERAPY FOR DM-1 MYOTONIC DYSTROPHY

CONJUGATED OLIGONUCLEOTIDES AND USES THEREOF

MUSCLE TARGETING COMPLEXES AND USES THEREOF FOR TREATING DYSTROPHIN...

NUCLEIC ACID MOLECULE AND ITS USE

ANTI-TRANSFERRIN RECEPTOR ANTIBODY-PMO CONJUGATES FOR INDUCING DMD...

USE OF SPLICE SWITCHING OLIGONUCLEOTIDES FOR EXON SKIPPING-MEDIATED...

MUSCLE TARGETING COMPLEXES AND USES THEREOF FOR TREATING DYSTROPHIN...

OLIGOMERIC COMPOUND FOR DYSTROPHIN RESCUE IN DMD PATIENTS THROUGHOU...

COMPOUNDS AND METHODS FOR MODULATING SCN1A EXPRESSION

ANTISENSE OLIGONUCLEOTIDE THERAPY FOR H3.3 K27M DIFFUSE MIDLINE GLI...

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

NUCLEIC ACID-POLYPEPTIDE COMPOSITIONS AND METHODS OF INDUCING EXON...

SNRNA TARGETING USH2A PRE-MRNA AND APPLICATION THEREOF

METHODS OF SYNTHESIZING OLIGONUCLEOTIDES USING TETHERED NUCLEOTIDES

NOVEL BICYCLIC NUCLEOSIDES AND OLIGOMERS PREPARED THEREFROM

METHOD

COMPOSITIONS COMPRISING EXON SKIPPING OLIGONUCLEOTIDE CONJUGATES FO...

ANTISENSE POLYNUCLEOTIDES TO INDUCE EXON SKIPPING AND METHOD OF TRE...