NUCLEIC ACID-POLYPEPTIDE COMPOSITIONS AND METHODS OF INDUCING EXON SKIPPING

• Main Authors: JOHNS RACHEL, LEVIN ARTHUR A, COCHRAN MICHAEL CARAMIAN, SHI YUNYU, DOPPALAPUDI VENKATA RAMANA, GEALL ANDREW JOHN, DARIMONT BEATRICE DIANA, BURKE ROB, HUANG HANHUA
• Format: Patent
• Language: Chinese; English
• Published: 13.10.2020
• Subjects: BEER; BIOCHEMISTRY; CHEMISTRY; COMPOSITIONS THEREOF; CULTURE MEDIA; ENZYMOLOGY; HUMAN NECESSITIES; HYGIENE; MEDICAL OR VETERINARY SCIENCE; METALLURGY; MICROBIOLOGY; MICROORGANISMS OR ENZYMES; MUTATION OR GENETIC ENGINEERING; PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS; SPIRITS; VINEGAR; WINE

Disclosed herein are molecules and pharmaceutical compositions that induce an insertion, deletion, duplication, or alteration in an incorrectly spliced mRNA transcript to induce exon skipping or exoninclusion. Also described herein include methods for treating a disease or disorder that comprises a...