Article
Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial.
Human gene therapy - 1 Oct 2008
Hauswirth William W, Aleman Tomas S, Kaushal Shalesh, Cideciyan Artur V, Schwartz Sharon B, Wang Lili, Conlon Thomas J, Boye Sanford L, Flotte Terence R, Byrne Barry J, Jacobson Samuel G
Abstract excerpt
Leber congenital amaurosis (LCA) is a group of autosomal recessive blinding retinal diseases that are incurable. One molecular form is caused by mutations in the RPE65 (retinal pigment epithelium-specific 65-kDa) gene. A recombinant adeno-associated virus serotype 2 (rAAV2) vector, altered to carry the human RPE65 gene (rAAV2-CBSB-hRPE65), restored vision in animal models with RPE65 deficiency. A clinical trial...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.