Success in sight: The eyes have it! Ocular gene therapy trials for LCA look promising

Gene Therapy

Volumn 15, Issue 17, 2008, Pages 1191-1192

  Bainbridge, J W ^a   Ali, R R ^a  

^a UNIVERSITY COLLEGE LONDON   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

COMPLEMENTARY DNA; EYE PROTEIN; PARVOVIRUS VECTOR; RPE65;

CLINICAL TRIAL; DISEASE MODEL; DISEASE SEVERITY; FOLLOW UP; GENE EXPRESSION; GENE REPLACEMENT THERAPY; GENE TARGETING; GENE TRANSFER; HUMAN; LEBER CONGENITAL AMAUROSIS; NONHUMAN; NOTE; PIGMENT EPITHELIUM; PRIORITY JOURNAL; PROMOTER REGION; PUPILLOMETRY; RETINA CELL; RETINA DEGENERATION; RETINA DETACHMENT; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; VISION; VISUAL ACUITY; VISUAL IMPAIRMENT;

CARRIER PROTEINS; CLINICAL TRIALS AS TOPIC; DEPENDOVIRUS; EYE PROTEINS; GENE EXPRESSION; GENE THERAPY; GENETIC VECTORS; OPTIC ATROPHIES, HEREDITARY; PIGMENT EPITHELIUM OF EYE; TRANSDUCTION, GENETIC;

EID: 50049136340     PISSN: 09697128     EISSN: 14765462     Source Type: Journal    
DOI: 10.1038/gt.2008.117     Document Type: Note

References (2)

1. Bainbridge JW, Smith AJ, Barker SS, Robbie S, Henderson R, Balaggan K et al. Effect of gene therapy on visual function in leber's congenital amaurosis. N Engl J Med 2008; 358: 2231-2239.
2. Maguire AM, Simonelli F, Pierce EA, Pugh Jr EN, Mingozzi F, Bennicelli J et al. Safety and efficacy of gene transfer for leber's congenital amaurosis. N Engl J Med 2008; 358: 2240-2248.