AAV-Mediated Gene Therapy for Choroideremia: Preclinical Studies in Personalized Models

PLoS ONE

Volumn 8, Issue 5, 2013, Pages

  Vasireddy, Vidyullatha ^a   Mills, Jason A ^b   Gaddameedi, Rajashekhar ^a   Basner Tschakarjan, Etiena ^b   Kohnke, Monika ^c   Black, Aaron D ^a   Alexandrov, Krill ^c   Zhou, Shangzhen ^b   Maguire, Albert M ^a,b   Chung, Daniel C ^a,b   Mac, Helen ^b   Sullivan, Lisa ^b   Gadue, Paul ^b   Bennicelli, Jeannette L ^a   French, Deborah L ^b   Bennett, Jean ^a,b  

^a UNIVERSITY OF PENNSYLVANIA   (United States)

^b CHILDREN S HOSPITAL OF PHILADELPHIA   (United States)

^c UNIVERSITY OF QUEENSLAND   (Australia)

Author keywords

[No Author keywords available]

Indexed keywords

ADENOVIRUS VECTOR; COMPLEMENTARY DNA; PARVOVIRUS VECTOR; RAB ESCORT PROTEIN 1; RAB ESCORT PROTEIN 1 COMPLEMENTARY DNA; RAB PROTEIN; RAB27A PROTEIN; RECOMBINANT PARVOVIRUS VECTOR; UNCLASSIFIED DRUG;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; APOPTOSIS; ARTICLE; BIOLOGICAL MODEL; CELL VIABILITY; CELLULAR DISTRIBUTION; CHOROIDEREMIA; CHOROIDEREMIA GENE; CONCENTRATION RESPONSE; CONTROLLED STUDY; DRUG EFFICACY; DRUG SAFETY; ENZYME ACTIVITY; FEMALE; GENE; GENETIC TRANSDUCTION; HISTOPATHOLOGY; HUMAN; HUMAN CELL; IN VITRO STUDY; IN VIVO STUDY; LOSS OF FUNCTION MUTATION; LYMPHOBLAST; MOLECULAR CLONING; MOUSE; NONHUMAN; PLURIPOTENT STEM CELL; PROTEIN EXPRESSION; PROTEIN FUNCTION; PROTEIN LOCALIZATION; PROTEIN PRENYLATION; PROTEIN TRANSPORT; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY;

ADAPTOR PROTEINS, SIGNAL TRANSDUCING; ANIMALS; CELL LINE; CHOROIDEREMIA; DEPENDOVIRUS; FEMALE; GENETIC THERAPY; HUMANS; INDIVIDUALIZED MEDICINE; MALE; MICE; PLASMIDS; PROTEIN TRANSPORT; RAB GTP-BINDING PROTEINS; SAFETY;

EID: 84877148806     PISSN: None     EISSN: 19326203     Source Type: Journal    
DOI: 10.1371/journal.pone.0061396     Document Type: Article

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