Article
Gene therapy for Leber congenital amaurosis: advances and future directions.
Graefe's archive for clinical and experimental ophthalmology = Albrecht von Graefes Archiv fur klinische und experimentelle Ophthalmologie - 1 Aug 2012
Hufnagel Robert B, Ahmed Zubair M, Corrêa Zélia M, Sisk Robert A
Abstract excerpt
BACKGROUND: Leber congenital amaurosis (LCA) is a congenital retinal dystrophy that results in significant and often severe vision loss at an early age. Comprehensive analysis of the genetic mutations and phenotypic correlations in LCA patients has allowed for significant improvements in understanding molecular pathways of photoreceptor degeneration and dysfunction. The purpose of this article is to review the...
Topics
- Animals
- Clinical Trials as Topic
- Dependovirus
- Gene Transfer Techniques
- Genetic Therapy
- Genetic Vectors
- Humans
- Leber Congenital Amaurosis
- Mutation
- cis-trans-Isomerases