Title

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

UMMS Affiliation

Gene Therapy Center; Department of Pediatrics

Publication Date

2007-09-11

Document Type

Article

Subjects

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.

DOI of Published Version

10.2217/14622416.8.9.1191

Source

Pharmacogenomics. 2007 Sep;8(9):1191-8. Link to article on publisher's site

Journal/Book/Conference Title

Pharmacogenomics

Related Resources

Link to Article in PubMed

PubMed ID

17924834

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