Cruz, Pedro E.Mueller, ChristianFlotte, Terence R.2022-08-232022-08-232007-09-112012-01-11Pharmacogenomics. 2007 Sep;8(9):1191-8. <a href="http://dx.doi.org/10.2217/14622416.8.9.1191">Link to article on publisher's site</a>1462-2416 (Linking)10.2217/14622416.8.9.119117924834https://hdl.handle.net/20.500.14038/43796In 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.en-USAnimalsDependovirusDisease Models, AnimalGene TherapyGenetic VectorsHumansTreatment Outcomealpha 1-Antitrypsin DeficiencyAllergy and ImmunologyGenetics and GenomicsPediatricsJournal Articlehttps://escholarship.umassmed.edu/peds_pulmonary/122441372peds_pulmonary/12