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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Veröffentlicht in:	Human gene therapy 2008-10, Vol.19 (10), p.979-990
Hauptverfasser:	HAUSWIRTH, William W, ALEMAN, Tomas S, JACOBSON, Samuel G, KAUSHAL, Shalesh, CIDECIYAN, Artur V, SCHWARTZ, Sharon B, LILI WANG, CONLON, Thomas J, BOYE, Sanford L, FLOTTE, Terence R, BYRNE, Barry J
Format:	Artikel
Sprache:	eng
Schlagworte:	Adeno-associated virus Adolescent Adult Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy Animals Applied cell therapy and gene therapy Biological and medical sciences Biotechnology Blindness - congenital Blindness - genetics Blindness - metabolism Blindness - pathology Blindness - therapy Care and treatment Carrier Proteins - genetics Carrier Proteins - metabolism Causes of cis-trans-Isomerases Dark Adaptation Dependovirus Eye Proteins - genetics Eye Proteins - metabolism Female Follow-Up Studies Fundamental and applied biological sciences. Psychology Gene therapy Genetic aspects Genetic Diseases, Inborn - genetics Genetic Diseases, Inborn - metabolism Genetic Diseases, Inborn - pathology Genetic Diseases, Inborn - therapy Genetic Therapy Genetic vectors Genetic Vectors - administration & dosage Genetic Vectors - genetics Genetic Vectors - metabolism Health. Pharmaceutical industry Humans Industrial applications and implications. Economical aspects Male Medical sciences Methods Mutation Retina - metabolism Retinal diseases Scientific Papers Transfusions. Complications. Transfusion reactions. Cell and gene therapy Vision, Ocular
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