Long-term effect of gene therapy on Leber's congenital amaurosis

New England Journal of Medicine

Volumn 372, Issue 20, 2015, Pages 1887-1897

  Bainbridge, J W B ^a,c   Mehat, M S ^a,c   Sundaram, V ^a,c   Robbie, S J ^a,c   Barker, S E ^a   Ripamonti, C ^a   Georgiadis, A ^a,c   Mowat, F M ^a,e   Beattie, S G ^a   Gardner, P J ^a   Feathers, K L ^f   Luong, V A ^a   Yzer, S ⁱ   Balaggan, K ^a,c   Viswanathan, A ^a   De Ravel, T J L ^g   Casteels, I ^h   Holder, G E ^a   Tyler, N ^b   Fitzke, F W ^a   Weleber, R G ^j   Nardini, M ^a,d   Moore, A T ^a,c   Thompson, D A ^f   Petersen Jones, S M ^e   Michaelides, M ^a,c   Van Den Born, L I ⁱ   Stockman, A ^a   Smith, A J ^a   Rubin, G ^a   Ali, R R ^a,f   more..

^a UNIVERSITY COLLEGE LONDON   (United Kingdom)

^b and Geomatic Engineering ^*  (United Kingdom)

^c MOORFIELDS EYE HOSPITAL   (United Kingdom)

^d DURHAM UNIVERSITY   (United Kingdom)

^e MICHIGAN STATE UNIVERSITY   (United States)

^f UNIVERSITY OF MICHIGAN   (United States)

^g UNIVERSITY OF LEUVEN   (Belgium)

^h UNIVERSITY HOSPITALS LEUVEN   (Belgium)

ⁱ ROTTERDAM EYE HOSPITAL   (Netherlands)

^j OREGON HEALTH AND SCIENCE UNIVERSITY   (United States)

more..

Author keywords

[No Author keywords available]

Indexed keywords

COMPLEMENTARY DNA; PARVOVIRUS VECTOR; PEPTIDES AND PROTEINS; RETINAL PIGMENT EPITHELIUM SPECIFIC PROTEIN 65; UNCLASSIFIED DRUG; CIS TRANS ISOMERASE; RETINOID ISOMEROHYDROLASE;

ADOLESCENT; ADULT; ARTICLE; CHILD; CLINICAL ARTICLE; COLOR VISION; CONTROLLED STUDY; DNA SEQUENCE; DOSE RESPONSE; DRUG ACTIVITY; DRUG EFFECT; DRUG EFFICACY; DRUG MEGADOSE; DRUG SAFETY; ELECTRORETINOGRAPHY; EYE DISEASE; GENE; GENE MUTATION; GENE THERAPY; HUMAN; IMMUNE RESPONSE; INFLAMMATION; INTRAOCULAR INFLAMMATION; LACRIMAL FLUID; LEBER CONGENITAL AMAUROSIS; LOW DRUG DOSE; MICROPERIMETRY; NONHUMAN; PERIMETRY; PHASE 1 CLINICAL TRIAL; PHASE 2 CLINICAL TRIAL; PRESCHOOL CHILD; PRIORITY JOURNAL; RETINAL SENSITIVITY; RETINAL THICKNESS; RPE65 GENE; SCHOOL CHILD; SIDE EFFECT; SPECTRAL SENSITIVITY; TREATMENT DURATION; TREATMENT OUTCOME; VIRAL GENE DELIVERY SYSTEM; VIRUS RECOMBINANT; VISION; VISUAL ACUITY; VISUAL FIELD; VISUAL SYSTEM PARAMETERS; ADMINISTRATION AND DOSAGE; ANIMAL; CLINICAL TRIAL; DEPENDOPARVOVIRUS; DISEASE COURSE; DISEASE MODEL; DOG; GENE VECTOR; GENETICS; MUTATION; PHOTORECEPTOR CELL; PHYSIOLOGY; RETINA; YOUNG ADULT;

ADOLESCENT; ANIMALS; CHILD; CIS-TRANS-ISOMERASES; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; DISEASE PROGRESSION; DNA, COMPLEMENTARY; DOGS; GENETIC THERAPY; GENETIC VECTORS; HUMANS; LEBER CONGENITAL AMAUROSIS; MUTATION; PHOTORECEPTOR CELLS, VERTEBRATE; RETINA; VISION, OCULAR; YOUNG ADULT;

EID: 84929353143     PISSN: 00284793     EISSN: 15334406     Source Type: Journal    
DOI: 10.1056/NEJMoa1414221     Document Type: Article

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