Broad Functional Correction of Molecular Impairments by Systemic Delivery of scAAVrh74-hSGSH Gene Delivery in MPS IIIA Mice

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作者： Duncan, F. Jason ^[1] ; Naughton, Bartholomew J. ^[2] ; Zaraspe, Kimberly ^[3] ; Murrey, Darren A. ; Meadows, Aaron S. ; Clark, Kelly Reed ; Newsom, David E. ; White, Peter ; Fu, Haiyan ; McCarty, Douglas M.

作者单位： Nationwide Childrens Hosp, Res Inst, Ctr Gene Therapy, Columbus, OH 43205 USA ^[1] Nationwide Childrens Hosp, Res Inst, Biomed Genet Core, Ctr Microbial Pathogenesis, Columbus, OH ^[2] Ohio State Univ, Dept Pediat, Coll Med & Publ Hlth, Columbus, OH 43210 USA ^[3]

发布时间 2016-01-15

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Molecular therapy

2015年23卷4期

638-647页

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Broad Functional Correction of Molecular Impairments by Systemic Delivery of scAAVrh74-hSGSH Gene Delivery in MPS IIIA Mice

Molecular therapy

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Molecular therapy

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Broad Functional Correction of Molecular Impairments by Systemic Delivery of scAAVrh74-hSGSH Gene Delivery in MPS IIIA Mice

Molecular therapy

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Molecular therapy

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