Age-dependent effects of RPE65 gene therapy for Leber’s congenital amaurosis: a phase 1 dose-escalation trial

BACKGROUND: Gene therapy has the potential to reverse disease or prevent further deterioration of vision in patients with incurable inherited retinal degeneration. We therefore did a phase 1 trial to assess the effect of gene therapy on retinal and visual function in children and adults with Leber’s...

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Publicado en:Lancet
Main Authors: Maguire, Albert M, High, Katherine A, Auricchio, Alberto, Wright, J Fraser, Pierce, Eric A, Testa, Francesco, Mingozzi, Federico, Bennicelli, Jeannette L, Ying, Gui-shuang, Rossi, Settimio, Fulton, Ann, Marshall, Kathleen A, Banfi, Sandro, Chung, Daniel C, Morgan, Jessica IW, Hauck, Bernd, Zelenaia, Olga, Zhu, Xiaosong, Raffini, Leslie, Coppieters, Frauke, De Baere, Elfride, Shindler, Kenneth S, Volpe, Nicholas J, Surace, Enrico M, Acerra, Carmela, Lyubarsky, Arkady, Redmond, T Michael, Stone, Edwin, Sun, Junwei, McDonnell, Jennifer Wellman, Leroy, Bart P, Simonelli, Francesca, Bennett, Jean
Formato: Artigo
Idioma:Inglês
Publicado: 2009
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Article
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC4492302/
https://ncbi.nlm.nih.gov/pubmed/19854499
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/S0140-6736(09)61836-5
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