Gene therapy for retinitis pigmentosa caused by MFRP mutations: Human phenotype and preliminary proof of concept

Human Gene Therapy

Volumn 23, Issue 4, 2011, Pages 367-376

  Dinculescu, Astra ^a   Estreicher, Jackie ^b   Zenteno, Juan C ^c   Aleman, Tomas S ^b   Schwartz, Sharon B ^b   Huang, Wei Chieh ^b   Roman, Alejandro J ^b   Sumaroka, Alexander ^b   Li, Qiuhong ^a   Deng, Wen Tao ^a   Min, Seok Hong ^a   Chiodo, Vince A ^a   Neeley, Andy ^a   Liu, Xuan ^a   Shu, Xinhua ^e   Matias Florentino, Margarita ^c,d   Buentello Volante, Beatriz ^c   Boye, Sanford L ^a   Cideciyan, Artur V ^b   Hauswirth, William W ^a   Jacobson, Samuel G ^b   more..

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

^b UNIVERSITY OF PENNSYLVANIA   (United States)

^c UNIVERSIDAD NACIONAL AUTÓNOMA DE MÉXICO   (Mexico)

^d INSTITUTE OF OPHTHALMOLOGY CONDE DE VALENCIANA   (Mexico)

^e GLASGOW CALEDONIAN UNIVERSITY   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

BETA ACTIN; PARVOVIRUS VECTOR; PROTEIN; PROTEIN MFRP; UNCLASSIFIED DRUG;

ADOLESCENT; ADULT; AGED; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CONTROLLED STUDY; DRUG DELIVERY SYSTEM; ELECTRORETINOGRAPHY; FEMALE; GENE; GENE MUTATION; GENE THERAPY; HISTOPATHOLOGY; HUMAN; INJECTION SITE; MFRP GENE; MOUSE; NONHUMAN; PHENOTYPE; PROMOTER REGION; PROTEIN EXPRESSION; RETINA CONE; RETINA DEGENERATION; RETINA ROD; RETINITIS PIGMENTOSA;

ADOLESCENT; ADULT; AGED; ANIMALS; CHILD; EYE PROTEINS; GENETIC THERAPY; GENETIC VECTORS; HUMANS; MEMBRANE PROTEINS; MICE; MICE, INBRED C57BL; MIDDLE AGED; MUTATION; PHENOTYPE; RETINITIS PIGMENTOSA;

EID: 84860187190     PISSN: 10430342     EISSN: 15577422     Source Type: Journal    
DOI: 10.1089/hum.2011.169     Document Type: Article

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