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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Αποθηκεύτηκε σε:
Λεπτομέρειες βιβλιογραφικής εγγραφής
Τόπος έκδοσης:Lancet
Κύριοι συγγραφείς: 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
Μορφή: Artigo
Γλώσσα:Inglês
Έκδοση: 2009
Θέματα:
Article
Διαθέσιμο Online: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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