Title
The promise of gene therapy for the treatment of alpha-1 antitrypsin deficiency
UMMS Affiliation
Gene Therapy Center; Department of Pediatrics
Date
9-11-2007
Document Type
Article
Medical Subject Headings
Animals; Dependovirus; Disease Models, Animal; Gene Therapy; Genetic Vectors; Humans; Treatment Outcome; alpha 1-Antitrypsin Deficiency
Disciplines
Allergy and Immunology | Genetics and Genomics | Pediatrics
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.
Rights and Permissions
Citation: Pharmacogenomics. 2007 Sep;8(9):1191-8. Link to article on publisher's site