Boucher R C
Department of Medicine, University of North Carolina at Chapel Hill 27589-7020.
Curr Opin Biotechnol. 1994 Dec;5(6):639-42. doi: 10.1016/0958-1669(94)90087-6.
A broad-based approach will be required for the development of new therapies for cystic fibrosis lung disease. Recently, rapid progress has been made in identifying and testing a number of gene transfer vectors, including adenoviral vectors and liposomes. Major problems, however, have been identified with respect to the efficiency of these systems. Preliminary studies suggest that small molecules (e.g. amelioride and UTP) may normalize the clearance of secretions from the cystic fibrosis lung. The concept of recombinant protein based therapy for cystic fibrosis has now been realized with the successful application of DNase in clinical trials.
开发囊性纤维化肺病的新疗法需要一种基础广泛的方法。最近，在鉴定和测试多种基因传递载体（包括腺病毒载体和脂质体）方面取得了快速进展。然而，已经发现这些系统在效率方面存在重大问题。初步研究表明，小分子（如氨甲酰脲和尿苷三磷酸）可能使囊性纤维化肺分泌物的清除恢复正常。随着脱氧核糖核酸酶在临床试验中的成功应用，基于重组蛋白的囊性纤维化治疗概念现已实现。
