公开(公告)号：US20250135032A1

公开(公告)日：2025-05-01

申请号：US18834040

申请日：2023-01-30

Applicant: TEMPLE UNIVERSITY - OF THE COMMONWEALTH SYSTEM OF HIGHER EDUCATION

Inventor： Kamel KHALILI ,  Chen CHEN ,  Shuren LIAO ,  Hong LIU

IPC: A61K48/00 ,  A61K38/46 ,  C12N15/11 ,  C12N15/86 ,  C12Q1/6883

Abstract: Compositions include gene-editing complexes for the treatment of myopathies, cancer and neurodegenerative diseases. Specifically, the disclosure provides methods of identifying in a subject's biological sample, ‘at least one Bcl2-associated anthanogene 3 (BAGS) genetic mutation as compared to a control BAGS nucleic acid sequence, and administering to the subject a therapeutically effective amount of a gene-editing complex, wherein the gene-editing complex corrects the bag3 mutation to a wild-type bag3, thereby, treating the subject.