The promise of gene therapy for the treatment of alpha-1 antitrypsin deficiency.
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.
Cruz
Pedro E
PE
University of Florida, Nephrology Division, College of Medicine, FL, USA.
Mueller
Christian
C
Flotte
Terence R
TR
D000818:Animals; D000229:Dependovirus / Q000235:genetics; D004195:Disease Models, Animal; D015316:Genetic Therapy / Q000379:methods* / Q000639:trends; D005822:Genetic Vectors; D006801:Humans; D016896:Treatment Outcome; D019896:alpha 1-Antitrypsin Deficiency / Q000235:genetics* / Q000628:therapy*