Extended Data Fig. 6: Normal growth of GRMD dogs randomized to AAV-µUtro as evidence against immune-mediated myositis.
From: Non-immunogenic utrophin gene therapy for the treatment of muscular dystrophy animal models
a, Individual weights of dogs randomized to the highest doses (3.16 × 1013 vg kg−1) of AAV-cU (AAV-μUtro) without immunosuppression (F[b] AAV-cU, F[c] AAV-cU), as well as relevant controls including littermates randomized to PBS and other littermate carrier females (F-carrier3, F-carrier4, F-carrier6) and non-littermate GRMD males (M-GRMD7, M-GRMD8) and females (F-GRMD9, F-GRMD10). Also included for comparison are relevant weights of previously reported GRMD females receiving human μDystro (F[e] AAV-hD, F[f] AAV-hD) showing rapid weight loss immediately prior to euthanasia and necropsy showing signs of systemic myositis14. b, Representative results of PCR assays for GRMD genotype. c, Graphical depiction of Animal ID#, creatine kinase (CK) levels at birth, GRMD status by PCR, and weight at two time points for littermates of AAV-μUtro injected dogs.
