Gene therapy for alpha 1-antitrypsin deficiency.
Alpha 1-antitrypsin deficiency is associated with pulmonary emphysema and liver cirrhosis. The pathogenesis and treatment is reviewed with particular emphasis on gene therapy for emphysema. The technology of gene transfer using viruses and liposomes is developing fast and animal experiments have confirmed the feasibility of gene therapy for alpha 1-antitrypsin deficiency (AATD). So far only subtherapeutic levels of protein have been produced but human trials are starting and progress can be expected.
Royal Brompton Hospital, London, UK.
D M
DM
Geddes
M A
MA
Beckles
D000818:Animals; D015316:Genetic Therapy; D005822:Genetic Vectors; D006801:Humans; D011656:Pulmonary Emphysema / Q000628:therapy; D019896:alpha 1-Antitrypsin Deficiency