Gene therapy

Gene therapy is the newest approach to the treatment of pulmonary disease. It can be defined as the introduction of nucleic acid sequences into cells for the purpose of altering a disease. Cystic fibrosis or alpha (1)-antitry psin deficiency are diseases associated with single gene defects and repres ent the obvious rationale for gene therapy in order to replace the defectiv e or absent gene. Chronic acquired respiratory disorders such as COPD, asth ma or interstitial lung diseases are considered to be the product of a vari ety of endogenous (polygenic) and exogenous influences and less obviously a ssociated with gene replacement therapy. These chronic inflammatory conditi ons likely arise from an imbalance between destructive and protective mecha nisms, such that transient gene therapy can be useful to reconstitute the i mpaired balance by short-term over-expression of protective genes or suppre ssion of damaging genes.