Human retinal gene therapy for Leber congenital amaurosis shows advancing retinal degeneration despite enduring visual improvement

Proceedings of the National Academy of Sciences of the United States of America

Volumn 110, Issue 6, 2013, Pages

  Cideciyan, Artur V ^a   Jacobson, Samuel G ^a   Beltran, William A ^b   Sumaroka, Alexander ^a   Swider, Malgorzata ^a   Iwabe, Simone ^b   Roman, Alejandro J ^a   Olivares, Melani B ^a   Schwartz, Sharon B ^a   Komáromy, András M ^b,c   Hauswirth, William W ^d   Aguirre, Gustavo D ^b  

^a UNIVERSITY OF PENNSYLVANIA   (United States)

^b UNIVERSITY OF PENNSYLVANIA   (United States)

^c MICHIGAN STATE UNIVERSITY   (United States)

^d UNIVERSITY OF FLORIDA COLLEGE OF MEDICINE   (United States)

Author keywords

Neurodegeneration; Outer nuclear layer; Retinal structure

Indexed keywords

MEMBRANE PROTEIN; RETINAL PIGMENT EPITHELIUM SPECIFIC PROTEIN 65 KDA; UNCLASSIFIED DRUG;

ADOLESCENT; ADULT; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CHILD; CLINICAL ARTICLE; CONTROLLED STUDY; ELECTRORETINOGRAPHY; GENE EXPRESSION; GENE LOCUS; GENE MUTATION; GENE THERAPY; HUMAN; LEBER CONGENITAL AMAUROSIS; NONHUMAN; PHOTORECEPTOR; PRIORITY JOURNAL; RETINA DEGENERATION; RETINAL THICKNESS; SCHOOL CHILD; SHORT COURSE THERAPY; VISION;

ADOLESCENT; ADULT; ANIMALS; CHILD; CHILD, PRESCHOOL; CIS-TRANS-ISOMERASES; DISEASE MODELS, ANIMAL; DOG DISEASES; DOGS; GENETIC THERAPY; HUMANS; LEBER CONGENITAL AMAUROSIS; MUTATION; PHOTORECEPTOR CELLS, VERTEBRATE; RETINAL DEGENERATION; TIME FACTORS; VISION, OCULAR; YOUNG ADULT;

EID: 84873453664     PISSN: 00278424     EISSN: 10916490     Source Type: Journal    
DOI: 10.1073/pnas.1218933110     Document Type: Article

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