Gene Therapy for Leber Congenital Amaurosis Caused by RPE65 Mutations: Safety and Efficacy in 15 Children and Adults Followed Up to 3 Years

OBJECTIVE To determine the safety and efficacy of subretinal gene therapy in the RPE65 form of Leber congenital amaurosis using recombinant adeno-associated virus 2 (rAAV2) carrying the RPE65 gene. DESIGN Open-label, dose-escalation phase I study of 15 patients (range, 11-30 years of age) evaluated...

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Veröffentlicht in:	Archives of ophthalmology (1960) 2012-01, Vol.130 (1), p.9-24
Hauptverfasser:	Jacobson, Samuel G, Cideciyan, Artur V, Ratnakaram, Ramakrishna, Heon, Elise, Schwartz, Sharon B, Roman, Alejandro J, Peden, Marc C, Aleman, Tomas S, Boye, Sanford L, Sumaroka, Alexander, Conlon, Thomas J, Calcedo, Roberto, Pang, Ji-Jing, Erger, Kirsten E, Olivares, Melani B, Mullins, Cristina L, Swider, Malgorzata, Kaushal, Shalesh, Feuer, William J, Iannaccone, Alessandro, Fishman, Gerald A, Stone, Edwin M, Byrne, Barry J, Hauswirth, William W
Format:	Artikel
Sprache:	eng
Schlagworte:	Adeno-associated virus 2 Adolescent Adult Biological and medical sciences Carrier Proteins - genetics Child cis-trans-Isomerases Dependovirus - genetics Eye diseases Eye Proteins - genetics Female Follow-Up Studies Gene therapy Genetic Therapy - adverse effects Genetic Therapy - methods Genetic Vectors Humans Injections, Intraocular Leber Congenital Amaurosis - genetics Leber Congenital Amaurosis - physiopathology Leber Congenital Amaurosis - therapy Male Medical sciences Miscellaneous Mutation Ophthalmology Photic Stimulation Photoreceptor Cells, Vertebrate - physiology Psychomotor Performance - physiology Pupil - physiology Retinopathies Risk assessment Tomography, Optical Coherence Treatment Outcome Visual Acuity - physiology Visual Fields - physiology Young Adult
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