Fig. 4. Transduction of striated muscle in adult GAA-KO mice with intravenously administered AAV2/7 or AAV2/9 vector containing the MHCK7 regulatory cassette.
(A) GAA activity in the indicated striated muscles, 18 weeks following administration of AAV-MHCK7hGAApA (5×1011 vp) pseudotyped as AAV2/7 (n=5) and AAV2/9 (n=5). Mock-treated GAA-KO mice were negative controls (PBS; n=4). (B) Glycogen content for GAA-KO mice in A. (C) Vector genome quantitation with realtime PCR for GAA-KO mice in A. (D) Ratio of GAA activity to vector genome quantity for GAA-KO mice in A. Mean +/- s.d. shown. P<0.05 (*) and P<0.001 (**) indicated for the values for each group of mice following AAV2/9 injection, in comparison to the group receiving the AAV2/7 vector.
