TY  - GEN
AU  - Maguire,Albert M
AU  - High,Katherine A
AU  - Auricchio,Alberto
AU  - Wright,J Fraser
AU  - Pierce,Eric A
AU  - Testa,Francesco
AU  - Mingozzi,Federico
AU  - Bennicelli,Jeannette L
AU  - Ying,Gui-shuang
AU  - Rossi,Settimio
AU  - Fulton,Ann
AU  - Marshall,Kathleen A
AU  - Banfi,Sandro
AU  - Chung,Daniel C
AU  - Morgan,Jessica I W
AU  - Hauck,Bernd
AU  - Zelenaia,Olga
AU  - Zhu,Xiaosong
AU  - Raffini,Leslie
AU  - Coppieters,Frauke
AU  - De Baere,Elfride
AU  - Shindler,Kenneth S
AU  - Volpe,Nicholas J
AU  - Surace,Enrico M
AU  - Acerra,Carmela
AU  - Lyubarsky,Arkady
AU  - Redmond,T Michael
AU  - Stone,Edwin
AU  - Sun,Junwei
AU  - McDonnell,Jennifer Wellman
AU  - Leroy,Bart P
AU  - Simonelli,Francesca
AU  - Bennett,Jean
TI  - Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial
SN  - 1474-547X
PY  - 2009///1123
KW  - Adolescent
KW  - Adult
KW  - Age Factors
KW  - Blindness
KW  - congenital
KW  - Carrier Proteins
KW  - genetics
KW  - Child
KW  - Dark Adaptation
KW  - Dependovirus
KW  - Disease Progression
KW  - Dose-Response Relationship, Drug
KW  - Electroretinography
KW  - Eye Proteins
KW  - Female
KW  - Genetic Therapy
KW  - methods
KW  - Genetic Vectors
KW  - Humans
KW  - Injections
KW  - Male
KW  - Mutation
KW  - Nystagmus, Physiologic
KW  - Optic Atrophy, Hereditary, Leber
KW  - diagnosis
KW  - Safety
KW  - Treatment Outcome
KW  - Visual Acuity
KW  - Young Adult
KW  - cis-trans-Isomerases
N1  - Publication Type: Clinical Trial, Phase I; Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
UR  - https://doi.org/10.1016/S0140-6736(09)61836-5
ER  -