The promise of gene therapy for the treatment of alpha-1 antitrypsin deficiency
Gene Therapy Center; Department of Pediatrics
Medical Subject Headings
Animals; Dependovirus; Disease Models, Animal; Gene Therapy; Genetic Vectors; Humans; Treatment Outcome; alpha 1-Antitrypsin Deficiency
Allergy and Immunology | Genetics and Genomics | Pediatrics
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.
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Citation: Pharmacogenomics. 2007 Sep;8(9):1191-8. Link to article on publisher's site