Treatment of Leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: Short-term results of a phase I trial

Human Gene Therapy

Volumn 19, Issue 10, 2008, Pages 979-990

  Hauswirth, William W ^a,b   Aleman, Tomas S ^c   Kaushal, Shalesh ^a   Cideciyan, Artur V ^c   Schwartz, Sharon B ^c   Wang, Lili ^d   Conlon, Thomas J ^a   Boye, Sanford L ^a   Flotte, Terence R ^e   Byrne, Barry J ^a,b   Jacobson, Samuel G ^c  

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

^b UNIVERSITY OF FLORIDA   (United States)

^c UNIVERSITY OF PENNSYLVANIA   (United States)

^d UNIVERSITY OF PENNSYLVANIA   (United States)

^e UNIVERSITY OF MASSACHUSETTS MEDICAL SCHOOL   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

PARVOVIRUS VECTOR;

ADULT; ARTICLE; CASE REPORT; CLINICAL TRIAL; DRUG SAFETY; FEMALE; GENE MUTATION; HUMAN; LEBER CONGENITAL AMAUROSIS; MALE; OPTICAL COHERENCE TOMOGRAPHY; PHASE 1 CLINICAL TRIAL; PIGMENT EPITHELIUM; RETINA FOVEA; VISUAL ACUITY;

ADOLESCENT; ADULT; ANIMALS; BLINDNESS; CARRIER PROTEINS; DARK ADAPTATION; DEPENDOVIRUS; EYE PROTEINS; FEMALE; FOLLOW-UP STUDIES; GENE THERAPY; GENETIC DISEASES, INBORN; GENETIC VECTORS; HUMANS; MALE; MUTATION; RETINA; VISION, OCULAR;

ADENO-ASSOCIATED VIRUS; ANIMALIA;

EID: 54949104686     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/hum.2008.107     Document Type: Article

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