NUCLEIC ACID-POLYPEPTIDE COMPOSITIONS AND METHODS OF INDUCING EXON SKIPPING

Abstract: 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 exon inclusion. also described herein include methods for treating a disease or disorder that comprises a molecule or a pharmaceutical composition that induces an insertion, deletion, duplication, or alteration in an incorrectly spliced mrna transcript to induce exon skipping or exon inclusion.

Inventor(s): Arthur A. LEVIN, Andrew John GEALL, Venkata Ramana DOPPALAPUDI, Michael Caramian COCHRAN, Hanhua HUANG, Rob BURKE

CPC Classification: A61K31/7088 (PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES (devices or methods specially adapted for bringing pharmaceutical products into particular physical or administering forms ; chemical aspects of, or use of materials for deodorisation of air, for disinfection or sterilisation, or for bandages, dressings, absorbent pads or surgical articles ; soap compositions ))

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