Therapy with PPMOs leads to a comprehensive distribution in muscle tissue and efficacy in the mouse model and in cynomolgus macaques: a therapeutic platform for Duchenne muscular dystrophy

L Gan, L Wu, M Yao,JA Wood, CM Treleaven, NL Estrella,BM Wentworth, JS Charleston,JV Rutkowski,GJ Hanson, A Müller-York,MA Passini

Nervenheilkunde24. Kongress des Medizinisch-Wissenschaftlichen Beirates der Deutschen Gesellschaft für Muskelkranke (DGM) e.V.（2019）

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摘要

Duchenne muscular dystrophy (DMD) is triggered by mutations in the DMD gene. Exon skipping is a therapy strategy at DMD. Appropriately designed morpho-oligomers (PMOs) can cause exon skipping and the formation of an internally truncated dystrophin protein. Approximately 80% of DMD patients have genotypes that are accessible for exon skipping.

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关键词

muscular dystrophy,cynomolgus macaques,ppmos,muscle tissue

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