Dystrophin immunity after gene therapy for Duchenne's muscular dystrophy
CG Bönnemann, BA Belluscio, S Braun… - … England Journal of …, 2023 - Mass Medical Soc
CG Bönnemann, BA Belluscio, S Braun, C Morris, T Singh, F Muntoni
New England Journal of Medicine, 2023•Mass Medical SocDystrophin Immunity after Gene Therapy for DMD Five boys with Duchenne's muscular
dystrophy received three different AAV products. Serious adverse reactions consistent with
transgene expression occurred, and laboratory findings suggested cytotoxic T-cell immune
responses.
dystrophy received three different AAV products. Serious adverse reactions consistent with
transgene expression occurred, and laboratory findings suggested cytotoxic T-cell immune
responses.
Dystrophin Immunity after Gene Therapy for DMD
Five boys with Duchenne’s muscular dystrophy received three different AAV products. Serious adverse reactions consistent with transgene expression occurred, and laboratory findings suggested cytotoxic T-cell immune responses.
The New England Journal Of Medicine