Abstract
Disclosed herein are CRISPR/Cas-based base editing compositions and methods for treating Duchenne Muscular Dystrophy by restoring dystrophin function.
Full Text
What is claimed is:
Disclosed herein are CRISPR/Cas-based base editing compositions and methods for treating Duchenne Muscular Dystrophy by restoring dystrophin function.
Timeline
Filed
04/29/2026Published
08/20/2026Granted
Not AvailableIPC Codes(5)
C12N 15/11:DNA or RNA fragments; Modified forms thereof (DNA or RNA not used in recombinant technology C07H 21/00)
A61K 31/713:Double-stranded nucleic acids or oligonucleotides
A61K 38/00:Medicinal preparations containing peptides (peptides containing beta-lactam rings A61K 31/00; cyclic dipeptides not having in their molecule any other peptide link than those which form their ring, e.g. piperazine-2,5-diones, A61K 31/00; ergoline-based peptides A61K 31/48; containing macromolecular compounds having statistically distributed amino acid units A61K 31/74; medicinal preparations containing antigens or antibodies A61K 39/00; medicinal preparations characterised by the non-active ingredients, e.g. peptides as drug carriers, A61K 47/00)