Gene therapy for cystic fibrosis: steady progress, should do well

作者：Eric W.F.W. Alton, DM Geddes · 发表于：European Respiratory Journal · 年份：1997 · DOI：10.1183/09031936.97.10020257 · 被引用次数：4 · 研究领域：Virus-based gene therapy research、Cystic Fibrosis Research Advances、Energy Harvesting in Wireless Networks

In the early 1990s, when clinical gene therapy trials were initiated for a number of diseases, including cystic fibrosis (CF), there was great expectation both amongst the general public and in some quarters of the scientific and medical profession that this new form of treatment would revolutionize medicine. Recently, a number of articles have been published, with respect to a spectrum of diseases, showing less than encouraging data with regard to such trials [1‐5]. This has been accompanied by a flurry of adverse publicity for gene therapy. The aim of this editorial is to suggest that neither the initial overexpectation, nor the present downturn of opinion, have been justified in considering this new form of treatment. It is well-recognized that a new therapeutic product takes a decade or longer to move from the laboratory to the patient. Gene therapy for CF has had a life span of only half this time, and is already in phase I clinical trials. We would suggest that the pace of progress has, in fact, been exceptional in this field, and it is likely that this will continue to be the case, if not distracted by unreasonable expectations or predictable downturns. Initial studies in vitro [6, 7], and subsequently in animal models in vivo [8, 9], have demonstrated the feasibility of gene therapy for CF. Whilst a number of vector systems are available to allow expression of a normal copy of the cystic fibrosis transmembrane (conductance) regulator (CFTR) gene, two (adenoviruses and...