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Gene therapy using self-complementary Y733F capsid mutant AAV2/8 restores vision in a model of early onset Leber congenital amaurosis.
Ku, Cristy A; Chiodo, Vince A; Boye, Sanford L; Goldberg, Andrew F X; Li, Tiansen; Hauswirth, William W; Ramamurthy, Visvanathan.
Affiliation
  • Ku CA; Center for Neuroscience, West Virginia University, Morgantown, WV 26505, USA.
Hum Mol Genet ; 20(23): 4569-81, 2011 Dec 01.
Article in En | MEDLINE | ID: mdl-21880665
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Vision, Ocular / Genetic Therapy / Capsid / Dependovirus / Leber Congenital Amaurosis / Mutation Limits: Animals / Humans Language: En Journal: Hum Mol Genet Journal subject: BIOLOGIA MOLECULAR / GENETICA MEDICA Year: 2011 Document type: Article Affiliation country: United States Country of publication: United kingdom
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Vision, Ocular / Genetic Therapy / Capsid / Dependovirus / Leber Congenital Amaurosis / Mutation Limits: Animals / Humans Language: En Journal: Hum Mol Genet Journal subject: BIOLOGIA MOLECULAR / GENETICA MEDICA Year: 2011 Document type: Article Affiliation country: United States Country of publication: United kingdom