P.193 Phosphoryl guanidine-containing backbone modifications enhance exon skipping, dystrophin restoration and survival in a severe mouse model for DMD

X. Hu, P. Kandasamy, G. McClorey, M. Shimizu, N. Kothari, N. Iwamoto, M. Byrne,F. Liu, K. Longo, J. Oieni,C. Shivalila,C. Rinaldi,H. Yang, M. Wood, C. Vargeese

Neuromuscular Disorders(2022)

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关键词
dystrophin restoration,exon skipping,severe mouse model,guanidine-containing
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