Restoration of full-length dystrophin expression through exon skipping in Duchenne muscular dystrophy patients with single exon duplications

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作者
Nicolau, S. [1 ]
Malhotra, J. [2 ]
Iammarino, M. [1 ]
Reash, N. [1 ]
Lowes, L. [1 ]
Flanigan, K. [1 ,3 ]
机构
[1] Nationwide Childrens Hosp, Columbus, OH USA
[2] Sarepta Therapeut, Cambridge, MA USA
[3] Ohio State Univ, Columbus, OH USA
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R74 [神经病学与精神病学];
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LSVP.32
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页数:1
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