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

Maguire, Albert M

Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial. [electronic resource] - Lancet (London, England) Nov 2009 - 1597-605 p. digital

Publication Type: Clinical Trial, Phase I; Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't

1474-547X

10.1016/S0140-6736(09)61836-5 doi


Adolescent
Adult
Age Factors
Blindness--congenital
Carrier Proteins--genetics
Child
Dark Adaptation
Dependovirus--genetics
Disease Progression
Dose-Response Relationship, Drug
Electroretinography
Eye Proteins--genetics
Female
Genetic Therapy--methods
Genetic Vectors--genetics
Humans
Injections
Male
Mutation--genetics
Nystagmus, Physiologic
Optic Atrophy, Hereditary, Leber--diagnosis
Safety
Treatment Outcome
Visual Acuity
Young Adult
cis-trans-Isomerases