Clinical gene therapy for the treatment of RPE65-associated Leber congenital amaurosis

Expert Opinion on Biological Therapy

Volumn 11, Issue 3, 2011, Pages 429-439

  Stein, Linda ^a   Roy, Kamolika ^a   Lei, Lei ^a   Kaushal, Shalesh ^a  

^a UNIVERSITY OF MASSACHUSETTS MEDICAL SCHOOL   (United States)

Author keywords

LCA; rAAV; RPE65 mutation; vision function

Indexed keywords

ADENOVIRUS VECTOR; RECOMBINANT ADENO ASSOCIATED VIRUS TYPE 2 RETINAL PIGMENT EPITHELIUM SPECIFIC 65 PROTEIN; RETINOID; UNCLASSIFIED DRUG; VISUAL PIGMENT;

ADENOVIRUS; ARTICLE; CLINICAL TRIAL; FLUSHING; HEADACHE; HUMAN; LEBER CONGENITAL AMAUROSIS; NONHUMAN; NYSTAGMUS; PHOTOPHOBIA; PIGMENT EPITHELIUM; PUPIL REFLEX; SIDE EFFECT; TRIACYLGLYCEROL BLOOD LEVEL; VIRAL GENE THERAPY;

ANIMALS; CARRIER PROTEINS; DEPENDOVIRUS; EVIDENCE-BASED MEDICINE; EYE PROTEINS; GENE THERAPY; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; HUMANS; LEBER CONGENITAL AMAUROSIS; RETINA; TRANSLATIONAL RESEARCH; TREATMENT OUTCOME; VISION, OCULAR;

ADENO-ASSOCIATED VIRUS;

EID: 79851493830     PISSN: 14712598     EISSN: None     Source Type: Journal    
DOI: 10.1517/14712598.2011.557358     Document Type: Article

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