COMPOSITIONS AND METHODS OF USE THEREOF FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY

• Main Author: Jaskula-Ranga, Vinod
• Format: Patent
• Language: English
• Published: 11.04.2024
• 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; ORGANIC CHEMISTRY; PEPTIDES; PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS; SPIRITS; VINEGAR; WINE

The present disclosure provides, in part, a method of treating subjects with Duchenne muscular dystrophy (DMD) via gene editing approaches that induce exon deletion(s) to restore the reading frame of the dystrophin gene, thereby restoring dystrophin protein activity. The invention also provides comp...