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    Functional characterization of a recombinant adeno-associated virus 5-pseudotyped cystic fibrosis transmembrane conductance regulator vector

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    Authors
    Sirninger, Jeffrey
    Mueller, Christian
    Braag, Sofia A.
    Tang, Qiushi
    Yue, Hungwen
    Detrisac, Carol
    Ferkol, Thomas
    Guggino, William B.
    Flotte, Terence R.
    UMass Chan Affiliations
    Department of Pediatrics
    Gene Therapy Center
    Document Type
    Journal Article
    Publication Date
    2004-09-09
    Keywords
    Cystic Fibrosis
    Cystic Fibrosis Transmembrane Conductance Regulator
    Gene Expression
    Gene Therapy
    Genetic Vectors
    Pseudomonas aeruginosa
    Allergy and Immunology
    Pediatrics
    Respiratory Tract Diseases
    
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    Link to Full Text
    http://dx.doi.org/10.1089/hum.2004.15.832
    Abstract
    Despite extensive experience with recombinant adeno-associated virus (rAAV) 2 vectors in the lung, gene expression has been low in the context of cystic fibrosis (CF) gene therapy, where the large size of the cystic fibrosis transmembrane conductance regulator (CFTR) coding sequence has prompted the use of compact endogenous promoter elements. We evaluated the possibility that gene expression from recombinant adeno-associated virus (rAAV) could be improved by using alternate AAV capsid serotypes that target different cell-surface receptors (i.e., rAAV5) and/or using stronger promoters. The relative activities of the cytomegalovirus (CMV) Rous sarcoma virus (RSV) promoter, the CMV enhancer/beta-actin (CB) promoter combination, and the CMV enhancer/RSV promoter hybrid were assessed in vitro in a CF bronchial cell line. The CB promoter was the most efficient. AAV capsid serotypes, rAAV2 and rAAV5, were also compared, and rAAV5 was found to be significantly more efficient. Based on these studies a rAAV5-CB-promoter-driven CFTR minigene vector was then used to correct the CF chloride transport defect in vitro, as well as the hyperinflammatory lung phenotype in Pseudomonas-agarose bead challenged CF mouse lungs in vivo. These studies provide functional characterization of a new version of rAAV-CFTR vectors.
    Source

    Hum Gene Ther. 2004 Sep;15(9):832-41.

    Permanent Link to this Item
    http://hdl.handle.net/20.500.14038/43853
    PubMed ID
    15353038
    Notes

    Christian Mueller is cited on this publication as Christian Muller. At the time of publication, Christian Mueller, Sofia Braag, Qiushi Tang and Terence Flotte were not yet affiliated with the University of Massachusetts Medical School.

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