Gene therapy for pulmonary diseases.

M. A. Rosenfeld, G. Ronald, R. G. Crystal

Research output: Contribution to journalReview article

15 Citations (Scopus)


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.

Original languageEnglish
Pages (from-to)677-680
Number of pages4
Issue number8
Publication statusPublished - Oct 1993


ASJC Scopus subject areas

  • Pathology and Forensic Medicine

Cite this

Rosenfeld, M. A., Ronald, G., & Crystal, R. G. (1993). Gene therapy for pulmonary diseases. Pathologie-biologie, 41(8), 677-680.