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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Hlavní autoři: 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
Médium: Artigo
Jazyk:Inglês
Vydáno: 2008
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Article
On-line přístup: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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