The promise of gene therapy for the treatment of alpha-1 antitrypsin deficiency
Document Type
Journal ArticlePublication Date
2007-09-11Keywords
AnimalsDependovirus
Disease Models, Animal
Gene Therapy
Genetic Vectors
Humans
Treatment Outcome
alpha 1-Antitrypsin Deficiency
Allergy and Immunology
Genetics and Genomics
Pediatrics
Metadata
Show full item recordAbstract
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 siteDOI
10.2217/14622416.8.9.1191Permanent Link to this Item
http://hdl.handle.net/20.500.14038/43796PubMed ID
17924834Related Resources
Link to Article in PubMedae974a485f413a2113503eed53cd6c53
10.2217/14622416.8.9.1191