Gene Transfer Clinical Trial for LGMD2E (Beta-sarcoglycan Deficiency) Using scAAVrh74.MHCK7.hSGCB
Status:
Recruiting
Trial end date:
2020-12-31
Target enrollment:
Participant gender:
Summary
The proposed clinical trial is the first-in-human, randomized, double-blinded, single dose
study of self-complementary AAVrh.74 carrying the human β-sarcoglycan gene under the control
of an MHCK7 promoter (scAAVrh74.MHCK7.hSGCB) delivered through a single systemic injection in
a peripheral vein of LGMD2E. Nine total subjects (6 treated, 3 placebo) will be enrolled.