Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial-Reference-Cited by-同舟云学术

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

Published:2009-11 Issue:9701 Volume:374 Page:1597-1605
ISSN:0140-6736
Container-title:The Lancet
language:en
Short-container-title:The Lancet

Author:

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

Publisher

Elsevier BV

Subject

General Medicine

Reference28 articles.

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2. Early-onset severe rod-cone dystrophy in young children with RPE65 mutations;Lorenz;Invest Ophthalmol Vis Sci,2000

3. Clinical and molecular genetics of Leber's congenital amaurosis: A multicenter study of Italian patients;Simonelli;Invest Ophthalmol Vis Sci,2007

4. Leber congenital amaurosis;Perrault;Mol Genet Metab,1999

5. Leber congenital amaurosis: genes, proteins and disease mechanisms;den Hollander;Prog Retinal Eye Res,2008

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