Reversal of Blindness in Animal Models of Leber Congenital Amaurosis Using Optimized AAV2-mediated Gene Transfer

We evaluated the safety and efficacy of an optimized adeno-associated virus (AAV; AAV2.RPE65) in animal models of the RPE65 form of Leber congenital amaurosis (LCA). Protein expression was optimized by addition of a modified Kozak sequence at the translational start site of hRPE65. Modifications in...

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Autori principali: Bennicelli, Jeannette, Wright, John Fraser, Komaromy, Andras, Jacobs, Jonathan B, Hauck, Bernd, Zelenaia, Olga, Mingozzi, Federico, Hui, Daniel, Chung, Daniel, Rex, Tonia S, Wei, Zhangyong, Qu, Guang, Zhou, Shangzhen, Zeiss, Caroline, Arruda, Valder R, Acland, Gregory M, Dell’Osso, Lou F, High, Katherine A, Maguire, Albert M, Bennett, Jean
Natura: Artigo
Lingua:Inglês
Pubblicazione: 2008
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Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC2842085/
https://ncbi.nlm.nih.gov/pubmed/18209734
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/sj.mt.6300389
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