rAAV1.tMCK.human-alpha-sarcoglycan

**rAAV1.tMCK.human-alpha-sarcoglycan** is an investigational, intramuscular recombinant adeno-associated virus serotype 1 gene-replacement therapy for limb-girdle muscular dystrophy type 2D, now commonly termed limb-girdle muscular dystrophy R3. The vector delivers a functional human **SGCA** transgene encoding alpha-sarcoglycan under control of the muscle-specific truncated muscle creatine kinase promoter. Expression of alpha-sarcoglycan is intended to restore the sarcoglycan complex within the dystrophin-associated glycoprotein complex and improve sarcolemmal stability in skeletal muscle. It was evaluated in a completed Phase 1 randomized, double-blind intramuscular gene-transfer study at Nationwide Children's Hospital, using injection into the extensor digitorum brevis muscle. ([clinicaltrials.gov](https://clinicaltrials.gov/study/NCT00494195))

Other names

rAAV1.tMCK.hSGCArAAV-1.tMCK.hSGCArAAV 1.tMCK.hSGCArAAV1.tMCK.hαSG