Visual acuity after retinal gene therapy for choroideremia

New England Journal of Medicine

Volumn 374, Issue 20, 2016, Pages 1996-1998

  Edwards, Thomas L ^a   Jolly, Jasleen K ^a   Groppe, Markus ^a   Barnard, Alun R ^a   Cottriall, Charles L ^b   Tolmachova, Tanya ^c   Black, Graeme C ^d   Webster, Andrew R ^e   Lotery, Andrew J ^f   Holder, Graham E ^e   Xue, Kanmin ^a   Downes, Susan M ^a   Simunovic, Matthew P ^a   Seabra, Miguel C ^c   MacLaren, Robert E ^a  

^a UNIVERSITY OF OXFORD   (United Kingdom)

^b OXFORD EYE HOSPITAL   (United Kingdom)

^c IMPERIAL COLLEGE LONDON   (United Kingdom)

^d UNIVERSITY OF MANCHESTER   (United Kingdom)

^e MOORFIELDS EYE HOSPITAL   (United Kingdom)

^f UNIVERSITY OF SOUTHAMPTON   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

PARVOVIRUS VECTOR;

CATARACT; CHM GENE; CHOROIDEREMIA; CLINICAL ARTICLE; CONTROLLED STUDY; ELECTROPHYSIOLOGY; GENE; GENE MUTATION; GENE THERAPY; HUMAN; LETTER; PHOTORECEPTOR CELL; PRIORITY JOURNAL; RETINAL PIGMENT EPITHELIUM; TREATMENT OUTCOME; VIRAL GENE DELIVERY SYSTEM; VISUAL ACUITY; CASE REPORT; COMPLICATION; RETINA;

CATARACT; CHOROIDEREMIA; GENETIC THERAPY; HUMANS; RETINA; VISUAL ACUITY;

EID: 84973472718     PISSN: 00284793     EISSN: 15334406     Source Type: Journal    
DOI: 10.1056/NEJMc1509501     Document Type: Letter

References (3)

1. Jacobson SG, Cideciyan AV, Roman AJ, et al. Improvement and decline in vision with gene therapy in childhood blindness. N Engl J Med 2015;372:1920-6.
2. Bainbridge JWB, Mehat MS, Sundaram V, et al. Long-term effect of gene therapy on Leber's congenital amaurosis. N Engl J Med 2015;372:1887-97.
3. MacLaren RE, Groppe M, Barnard AR, et al. Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial. Lancet 2014;383:1129-37.