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Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study |
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| Authors: | Cirak Sebahattin Arechavala-Gomeza Virginia Guglieri Michela Feng Lucy Torelli Silvia Anthony Karen Abbs Stephen Garralda Maria Elena Bourke John Wells Dominic J Dickson George Wood Matthew J A Wilton Steve D Straub Volker Kole Ryszard Shrewsbury Stephen B Sewry Caroline Morgan Jennifer E Bushby Kate Muntoni Francesco |
| Affiliation: | Dubowitz Neuromuscular Centre, UCL Institute of Child Health, London, UK. |
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| 本文献已被 PubMed 等数据库收录！ | |