In utero gene therapy rescues vision in a murine model of congenital blindness

Molecular Therapy

Volumn 9, Issue 2, 2004, Pages 182-188

  Dejneka, Nadine S ^a   Surace, Enrico M ^a   Aleman, Tomas S ^a   Cideciyan, Artur V ^a   Lyubarsky, Arkady ^a   Savchenko, Andrey ^a   Redmond, T Michael ^b   Tang, Waixing ^a   Wei, Zhangyong ^a   Rex, Tonia S ^a   Glover, Ernest ^a   Maguire, Albert M ^a   Pugh Jr, Edward N ^a   Jacobson, Samuel G ^a   Bennett, Jean ^a  

^a UNIVERSITY OF PENNSYLVANIA   (United States)

^b NATIONAL EYE INSTITUTE   (United States)

Author keywords

Adeno associated virus; Gene therapy; Genetic disease; In utero therapy; Mouse model; Neurodegeneration; Phototransduction; Retina; Retinal degeneration; Viral gene transfer

Indexed keywords

COMPLEMENTARY DNA; MEMBRANE PROTEIN; PARVOVIRUS VECTOR; PROTEIN RPE65; RHODOPSIN; UNCLASSIFIED DRUG;

ADENO ASSOCIATED VIRUS; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CONTROLLED STUDY; CYTOMEGALOVIRUS; FEMALE; FETUS; GENE MUTATION; GENETIC TRANSDUCTION; GESTATION PERIOD; HUMAN; LEBER CONGENITAL AMAUROSIS; MOUSE; NONHUMAN; PATHOGENESIS; PIGMENT EPITHELIUM; SEROTYPING; TREATMENT OUTCOME; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; VIRUS CAPSID; VIRUS TYPING; VISION;

ADENO-ASSOCIATED VIRUS; MURINAE;

EID: 10744230959     PISSN: 15250016     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.ymthe.2003.11.013     Document Type: Article

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