Alteration of splicing

Industry

CPC
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

Current Industry

C12N2320/33Alteration of splicing

Industries

Patents Grantslast 30 patents

Information Patent Grant
Oral delivery of antisense conjugates targeting PCSK9
- Patent number 12,291,708
- Issue date May 6, 2025
- CiVi Biopharma, Inc.
- Henrik Oerum
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Soluble interleukin-7 receptor (SIL7R) modulating therapy to treat...
- Patent number 12,247,206
- Issue date Mar 11, 2025
- Board of Regents, The University of Texas System
- Mariano A. Garcia-Blanco
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Methods of treating non-syndromic sensorineural hearing loss
- Patent number 12,226,493
- Issue date Feb 18, 2025
- Akouos, Inc.
- Emmanuel John Simons
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Pharmaceutical composition for treatment of diseases associated wit...
- Patent number 12,227,743
- Issue date Feb 18, 2025
- Osaka University
- Fumihiro Sanada
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
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...

Patents Applicationslast 30 patents

Information Patent Application
SPLICE-SWITCHING OLIGONUCLEOTIDES AND METHODS OF USE
- Publication number 20250136995
- Publication date May 1, 2025
- DUKE UNIVERSITY
- Jennifer Freedman
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
THERAPEUTIC APPLICATIONS OF CPF1-BASED GENOME EDITING
- Publication number 20250114482
- Publication date Apr 10, 2025
- DUKE UNIVERSITY
- Charles A. Gersbach
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
PRE-MRNA SLICE MODULATING PEPTIDE-CONJUGATED ANTISENSE THERAPEUTICS...
- Publication number 20250114464
- Publication date Apr 10, 2025
- The Governors of the University of Alberta
- Toshifumi YOKOTA
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
Exon skipping to treat Usher Syndrome
- Publication number 20250108129
- Publication date Apr 3, 2025
- Massachusetts Eye and Ear Infirmary
- Zheng-Yi Chen
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
ALLELE SPECIFIC SPLICE SWITCHING OLIGONUCLEOTIDES TARGETING PSEUDOE...
- Publication number 20250101419
- Publication date Mar 27, 2025
- SYDDANSK UNIVERSITET
- Brage Storstein ANDRESEN
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
ANTISENSE OLIGONUCLEOTIDES AND THEIR USE FOR TREATMENT OF NEURODEGE...
- Publication number 20250092392
- Publication date Mar 20, 2025
- Eisai R&D Management Co., Ltd.
- Vinod VATHIPADIEKAL
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
EXON SKIPPING OLIGOMERS AND OLIGOMER CONJUGATES FOR MUSCULAR DYSTROPHY
- Publication number 20250090569
- Publication date Mar 20, 2025
- Sarepta Therapeutics, Inc.
- Marco A. PASSINI
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
Precise Excisions of Portions of Exon 44, 50, and 53 for Treatment...
- Publication number 20250090686
- Publication date Mar 20, 2025
- VERTEX PHARMACEUTICALS INCORPORATED
- Tudor Fulga
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
EXON SKIPPING OLIGOMERS AND OLIGOMER CONJUGATES FOR MUSCULAR DYSTROPHY
- Publication number 20250092393
- Publication date Mar 20, 2025
- Sarepta Therapeutics, Inc.
- Frederick Joseph SCHNELL
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
METHODS AND COMPOSITIONS TO REGULATE MAPT SPLICING FOR MODELING AND...
- Publication number 20250084413
- Publication date Mar 13, 2025
- THE TRUSTEES OF COLUMBIA UNIVERSITY IN THE CITY OF NEW YORK
- Yocelyn RECINOS
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
Antisense Nucleic Acid
- Publication number 20250084411
- Publication date Mar 13, 2025
- Sumitomo Pharma Co., Ltd.
- Zhiqiu MAN
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
Antisense Nucleic Acids
- Publication number 20250084412
- Publication date Mar 13, 2025
- NIPPON SHINYAKU CO., LTD.
- Naoki Watanabe
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
ENGINEERED RNAS
- Publication number 20250084408
- Publication date Mar 13, 2025
- Shape Therapeutics Inc.
- Susan BYRNE
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
METHODS AND AGENTS FOR INCREASING RBM3 EXPRESSION
- Publication number 20250066777
- Publication date Feb 27, 2025
- FREIE UNIVERSITAT BERLIN
- Marco PREUßNER
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
OPA1 ANTISENSE OLIGOMERS FOR TREATMENT OF CONDITIONS AND DISEASES
- Publication number 20250059535
- Publication date Feb 20, 2025
- Stoke Therapeutics, Inc.
- Isabel Aznarez
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
RNA EDITING VIA RECRUITMENT OF SPLICEOSOME COMPONENTS
- Publication number 20250051764
- Publication date Feb 13, 2025
- Tacit Therapeutics, Inc.
- David A. Nelles
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
OLIGONUCLEOTIDE COMPOSITIONS AND METHODS OF USE THEREOF
- Publication number 20250051778
- Publication date Feb 13, 2025
- WAVE LIFE SCIENCES LTD.
- Michael John Byrne
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
COMPOSITIONS AND METHODS FOR MODULATING mRNA SPLICING
- Publication number 20250051780
- Publication date Feb 13, 2025
- Entrada Therapeutics, Inc.
- Ziqing QIAN
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
THERAPEUTICS FOR THE TREATMENT OF NEURODEGENERATIVE DISORDERS
- Publication number 20250051775
- Publication date Feb 13, 2025
- The United States of America, as represented by the Secretary, Department of...
- Pietro Fratta
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
COMPOSITIONS AND METHODS FOR MODULATING INTERFERON REGULATORY FACTO...
- Publication number 20250051393
- Publication date Feb 13, 2025
- Entrada Therapeutics, Inc.
- Ziqing QIAN
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
Antisense Compounds Targeting Genes Associated with Cystic Fibrosis
- Publication number 20250049838
- Publication date Feb 13, 2025
- ROSALIND FRANKLIN UNIVERSITY OF MEDICINE AND SCIENCE
- Michelle L. Hastings
- A01 - AGRICULTURE FORESTRY ANIMAL HUSBANDRY HUNTING TRAPPING FISHING
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...

Alteration of splicing

Industry

Parent Industries

Current Industry

Patents Grantslast 30 patents

Oral delivery of antisense conjugates targeting PCSK9

Soluble interleukin-7 receptor (SIL7R) modulating therapy to treat...

Methods of treating non-syndromic sensorineural hearing loss

Pharmaceutical composition for treatment of diseases associated wit...

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...

Patents Applicationslast 30 patents

SPLICE-SWITCHING OLIGONUCLEOTIDES AND METHODS OF USE

THERAPEUTIC APPLICATIONS OF CPF1-BASED GENOME EDITING

PRE-MRNA SLICE MODULATING PEPTIDE-CONJUGATED ANTISENSE THERAPEUTICS...

Exon skipping to treat Usher Syndrome

ALLELE SPECIFIC SPLICE SWITCHING OLIGONUCLEOTIDES TARGETING PSEUDOE...

ANTISENSE OLIGONUCLEOTIDES AND THEIR USE FOR TREATMENT OF NEURODEGE...

EXON SKIPPING OLIGOMERS AND OLIGOMER CONJUGATES FOR MUSCULAR DYSTROPHY

Precise Excisions of Portions of Exon 44, 50, and 53 for Treatment...

EXON SKIPPING OLIGOMERS AND OLIGOMER CONJUGATES FOR MUSCULAR DYSTROPHY

METHODS AND COMPOSITIONS TO REGULATE MAPT SPLICING FOR MODELING AND...

Antisense Nucleic Acid

Antisense Nucleic Acids

ENGINEERED RNAS

METHODS AND AGENTS FOR INCREASING RBM3 EXPRESSION

OPA1 ANTISENSE OLIGOMERS FOR TREATMENT OF CONDITIONS AND DISEASES

RNA EDITING VIA RECRUITMENT OF SPLICEOSOME COMPONENTS

OLIGONUCLEOTIDE COMPOSITIONS AND METHODS OF USE THEREOF

COMPOSITIONS AND METHODS FOR MODULATING mRNA SPLICING

THERAPEUTICS FOR THE TREATMENT OF NEURODEGENERATIVE DISORDERS

COMPOSITIONS AND METHODS FOR MODULATING INTERFERON REGULATORY FACTO...

Antisense Compounds Targeting Genes Associated with Cystic Fibrosis

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