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
Anti-transferrin receptor antibody-PMO conjugates for inducing DMD...
- Patent number 12,359,202
- Issue date Jul 15, 2025
- AVIDITY BIOSCIENCES, INC.
- Beatrice Diana Darimont
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Soluble interleukin-7 receptor (SIL7R) modulating therapy to treat...
- Patent number 12,359,204
- Issue date Jul 15, 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
CRISPR/Cas9-mediated exon-skipping approach for USH2A-associated us...
- Patent number 12,331,328
- Issue date Jun 17, 2025
- Massachusetts Eye and Ear Infirmary
- Eric A. Pierce
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Bispecific antisense oligonucleotides for dystrophin exon skipping
- Patent number 12,331,293
- Issue date Jun 17, 2025
- BIOMARIN TECHNOLOGIES B.V.
- Judith Christina Theodora van Deutekom
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
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

Patents Applicationslast 30 patents

Information Patent Application
EXON SKIPPING COMPOSITIONS FOR TREATING MUSCULAR DYSTROPHY
- Publication number 20250236869
- Publication date Jul 24, 2025
- Sarepta Therapeutics, Inc.
- Richard K. BESTWICK
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
COMPOSITIONS AND METHODS FOR TREATING A CONGENITAL EYE DISEASE
- Publication number 20250223587
- Publication date Jul 10, 2025
- Beam Therapeutics Inc.
- David Bryson
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
COMPOSITIONS AND METHODS FOR THE TREATMENT OF MUSCULAR DYSTROPHIES
- Publication number 20250215426
- Publication date Jul 3, 2025
- Astellas Gene Therapies, Inc.
- Yukari MORIYA
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
COMPOSITIONS AND USES FOR TREATMENT THEREOF
- Publication number 20250197860
- Publication date Jun 19, 2025
- 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 Application
PHOSPHORODIAMIDATE MORPHOLINO OLIGOMER CONJUGATES
- Publication number 20250195667
- Publication date Jun 19, 2025
- Sarepta Therapeutics, Inc.
- Gunnar J. Hanson
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
Antisense Nucleic Acids
- Publication number 20250188459
- Publication date Jun 12, 2025
- NIPPON SHINYAKU CO., LTD.
- Naoki Watanabe
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
METHODS OF TREATING GLAUCOMA
- Publication number 20250179490
- Publication date Jun 5, 2025
- PYC Therapeutics Limited
- Janya Grainok
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
COMPOUNDS AND METHODS FOR MODULATING SMN2
- Publication number 20250179493
- Publication date Jun 5, 2025
- Ionis Pharmaceuticals, Inc.
- Frank Rigo
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
EXON SKIPPING OLIGOMER CONJUGATES FOR MUSCULAR DYSTROPHY
- Publication number 20250179487
- Publication date Jun 5, 2025
- Sarepta Therapeutics, Inc.
- Frederick Joseph SCHNELL
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
ANTISENSE POLYNUCLEOTIDES TO INDUCE EXON SKIPPING AND METHOD OF TRE...
- Publication number 20250179495
- Publication date Jun 5, 2025
- The University of Chicago
- Elizabeth McNally
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
NUCLEIC ACID-POLYPEPTIDE COMPOSITIONS AND METHODS OF INDUCING EXON...
- Publication number 20250170260
- Publication date May 29, 2025
- Avidity Biosciences, Inc.
- Arthur A. LEVIN
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
ANTISENSE-INDUCED EXON2 INCLUSION IN ACID ALPHA-GLUCOSIDASE
- Publication number 20250171783
- Publication date May 29, 2025
- Sarepta Therapeutics, Inc.
- Stephen Donald WILTON
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
ANTISENSE OLIGOMERS AND METHODS OF USING THE SAME FOR TREATING DISE...
- Publication number 20250171779
- Publication date May 29, 2025
- Sarepta Therapeutics, Inc.
- Frederick Joseph Schnell
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
NUCLEIC ACID-POLYPEPTIDE COMPOSITIONS AND METHODS OF INDUCING EXON...
- Publication number 20250161344
- Publication date May 22, 2025
- Avidity Biosciences, Inc.
- Arthur A. LEVIN
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
ANTISENSE OLIGONUCLEOTIDES FOR INDUCING EXON SKIPPING AND METHODS O...
- Publication number 20250163414
- Publication date May 22, 2025
- THE UNIVERSITY OF WESTERN AUSTRALIA
- Stephen Donald Wilton
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
MULTIPLE EXON SKIPPING COMPOSITIONS FOR DMD
- Publication number 20250154507
- Publication date May 15, 2025
- Sarepta Therapeutics, Inc.
- Peter SAZANI
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
EXON SKIPPING COMPOSITIONS FOR TREATING MUSCULAR DYSTROPHY
- Publication number 20250145991
- Publication date May 8, 2025
- Sarepta Therapeutics, Inc.
- Richard K. BESTWICK
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
ENHANCED EXPRESSION OF ALTERNATIVE SPLICED UROMODULIN FOR THERAPEUT...
- Publication number 20250145993
- Publication date May 8, 2025
- THE TRUSTEES OF INDIANA UNIVERSITY
- Tarek Maurice Ashkar
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
Antisense Nucleic Acids
- Publication number 20250145997
- Publication date May 8, 2025
- Nippon Shinyaku co., Ltd.
- Tatsushi Wakayama
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
Ventricular Arrhythmias and Related Methods
- Publication number 20250143624
- Publication date May 8, 2025
- University of Massachusetts
- John Kevin Donahue
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
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...

Alteration of splicing

Industry

Parent Industries

Current Industry

Patents Grantslast 30 patents

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

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

CRISPR/Cas9-mediated exon-skipping approach for USH2A-associated us...

Bispecific antisense oligonucleotides for dystrophin exon skipping

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

Patents Applicationslast 30 patents

EXON SKIPPING COMPOSITIONS FOR TREATING MUSCULAR DYSTROPHY

COMPOSITIONS AND METHODS FOR TREATING A CONGENITAL EYE DISEASE

COMPOSITIONS AND METHODS FOR THE TREATMENT OF MUSCULAR DYSTROPHIES

COMPOSITIONS AND USES FOR TREATMENT THEREOF

PHOSPHORODIAMIDATE MORPHOLINO OLIGOMER CONJUGATES

Antisense Nucleic Acids

METHODS OF TREATING GLAUCOMA

COMPOUNDS AND METHODS FOR MODULATING SMN2

EXON SKIPPING OLIGOMER CONJUGATES FOR MUSCULAR DYSTROPHY

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

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

ANTISENSE-INDUCED EXON2 INCLUSION IN ACID ALPHA-GLUCOSIDASE

ANTISENSE OLIGOMERS AND METHODS OF USING THE SAME FOR TREATING DISE...

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

ANTISENSE OLIGONUCLEOTIDES FOR INDUCING EXON SKIPPING AND METHODS O...

MULTIPLE EXON SKIPPING COMPOSITIONS FOR DMD

EXON SKIPPING COMPOSITIONS FOR TREATING MUSCULAR DYSTROPHY

ENHANCED EXPRESSION OF ALTERNATIVE SPLICED UROMODULIN FOR THERAPEUT...

Antisense Nucleic Acids

Ventricular Arrhythmias and Related Methods

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...