Gene therapy for pulmonary diseases.

Publication Type	Review
Authors	Rosenfeld M, Ronald G, Crystal R
Journal	Pathol Biol (Paris)
Volume	41
Issue	8
Pagination	677-80
Date Published	10/01/1993
ISSN	0369-8114
Keywords	Cystic Fibrosis, Deficiency Diseases, Gene Transfer Techniques, Genetic Therapy, alpha 1-Antitrypsin Deficiency
Abstract	The common fatal hereditary disorders, alpha 1-antitrypsin (alpha 1AT) deficiency and cystic fibrosis (CF), are clinical models for the common lung diseases, emphysema and chronic bronchitis, respectively. Both are potentially amenable to therapeutic intervention by gene therapy, in which the relevant gene is used to modify cells of the affected individual. Although the gene therapy strategies for these diseases are conceptually quite different, a promising approach for both is the in vivo administration of a recombinant replication deficient adenovirus vector containing a normal copy of the abnormal gene. If the goal is to express the normal extracellular anti-protease alpha 1 AT, the route of administration could be directly into the lung, liver or vascular endothelium. If the goal is to express the normal transmembrane protein defective in CF epithelial cells, the new gene will need to be delivered directly to the affected cells. The feasibility of these approaches has been demonstrated in vitro and in vivo in experimental animals.
PubMed ID	8290311