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...

Ausführliche Beschreibung

Gespeichert in:
Bibliographische Detailangaben
Veröffentlicht in:Lancet
Hauptverfasser: 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
Format: Artigo
Sprache:Inglês
Veröffentlicht: 2009
Schlagworte:
Article
Online Zugang: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
Tags: Tag hinzufügen
Keine Tags, Fügen Sie den ersten Tag hinzu!

Ähnliche Einträge