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Immunologic investigations into transgene directed immune-mediated myositis following delandistrogene moxeparvovec gene therapy

Abstract Delandistrogene moxeparvovec is an rAAVrh74 vector-based gene transfer therapy that delivers a transgene encoding delandistrogene moxeparvovec micro-dystrophin, an engineered, functional form of dystrophin shown to stabilize or slow disease progression in DMD. It is approved in the US and i...

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Principais autores: Rachael A. Potter, Ida H. Moeller, Sohrab Khan, Hélène Haegel, Andreas Hollenstein, Guido Steiner, Christoph Wandel, Alexander P. Murphy, Damon R. Asher, Emanuel Palatinsky, Danielle A. Griffin, Stefanie Mason, Susan T. Iannaccone, Craig M. Zaidman, Louise R. Rodino-Klapac
Formato: Artigo
Idioma:Inglês
Publicado: Nature Portfolio 2025-01-01
Series:Scientific Reports
Assuntos:
AAV vector
Delandistrogene moxeparvovec
Duchenne muscular dystrophy
Dystrophin
Gene transfer therapy
Immune-mediated myositis
Acceso en liña:https://doi.org/10.1038/s41598-024-84077-w
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