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