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Dystrophin Immunity after Gene Therapy for Duchenne's Muscular Dystrophy

Carsten G. Boennemann,Beth A. Belluscio,Serge Braun,Carl Morris,Teji Singh,Francesco Muntoni

NEW ENGLAND JOURNAL OF MEDICINE（2023）

Cited 8|Views10
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Abstract
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.
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