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

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...

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Autors principals: 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.
Format: Artigo
Idioma:Inglês
Publicat: Mary Ann Liebert, Inc. 2008
Matèries:
Scientific Papers
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC2940541/
https://ncbi.nlm.nih.gov/pubmed/18774912
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2008.107
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