Dystrophin Immunity after Gene Therapy for Duchenne's Muscular Dystrophy

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

The New England journal of medicine（2023）

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摘要

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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关键词

muscular dystrophy,gene therapy,duchennes,immunity

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