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

Article, Other literature type English OPEN
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;
  • Publisher: Elsevier BV
  • Journal: Molecular Therapy,volume 16,issue 3,pages458-465 (issn: 1525-0016)
  • Related identifiers: doi: 10.1038/, pmc: PMC2842085
  • Subject: Molecular Biology | Genetics | Drug Discovery | Pharmacology | Molecular Medicine | Article
    mesheuropmc: eye diseases | viruses | genetic structures | sense organs

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 sit... View more
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