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    The promise of gene therapy for the treatment of alpha-1 antitrypsin deficiency

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    Authors
    Cruz, Pedro E.
    Mueller, Christian
    Flotte, Terence R.
    UMass Chan Affiliations
    Department of Pediatrics
    Gene Therapy Center
    Document Type
    Journal Article
    Publication Date
    2007-09-11
    Keywords
    Animals
    Dependovirus
    Disease Models, Animal
    Gene Therapy
    Genetic Vectors
    Humans
    Treatment Outcome
    alpha 1-Antitrypsin Deficiency
    Allergy and Immunology
    Genetics and Genomics
    Pediatrics
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    Link to Full Text
    http://dx.doi.org/10.2217/14622416.8.9.1191
    Abstract
    In the last 13 years, three gene therapy trials for the treatment of alpha-1 antitrypsin deficiency have been conducted. The first trial delivered plasmid encoding the alpha-1 antitrypsin cDNA to the nasal epithelium using cationic liposomes. The last two trials delivered recombinant adeno-associated vectors encoding the alpha-1 antitrypsin cDNA by intramuscular injection. In this review, the progress of ongoing clinical trials and new gene therapy technologies is discussed.
    Source
    Pharmacogenomics. 2007 Sep;8(9):1191-8. Link to article on publisher's site
    DOI
    10.2217/14622416.8.9.1191
    Permanent Link to this Item
    http://hdl.handle.net/20.500.14038/43796
    PubMed ID
    17924834
    Related Resources
    Link to Article in PubMed
    ae974a485f413a2113503eed53cd6c53
    10.2217/14622416.8.9.1191
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