Patent ID: 11925675
Assignee: SAREPTA THERAPEUTICS, INC.
Field: Pharmaceuticals (Chemistry)
Classification: CPC A  C | IPC A  C

Claim 1:
2. The method of claim 1, wherein the level of gamma-sarcoglycan gene expression in a cell of the subject is increased after administration of the rAAV as compared to the level of gamma-sarcoglycan gene expression before administration of the rAAV;
wherein the average rAAV copy number in a muscle cell of the treated subject is at least 0.01 copy per nucleus;
wherein the centralized nuclei percentage and/or the fibrosis is reduced in the muscle of the treated subject as compared to the level before administration of the rAAV;
wherein motor function is improved in said subject as compared to the motor function of said subject before administration of the rAAV;
wherein the North Star Assessment for Dysferlinopathies (NSAD) are increased in said subject as compared to the NSAD of said subject before administration of the rAAV; and/or
wherein the ALT and AST levels are reduced in said subject as compared to the ALT and AST levels of said subject before administration of the rAAV.