Willemot J-M, Sermet Gaudelus I, Lenoir G
Association ABCF Protéine, hôpital Necker Enfants Malades, Paris.
Ann Pharm Fr. 2003 Jul;61(4):253-8.
Since the cloning of the defective gene in cystic fibrosis, much has been learned on the function of CFTR and on the mechanisms regulating its expression. Based on the current understanding of the processes involved in lung disease progression, a number of approaches have been developed using gene therapy and pharmacological agents. Several of these agents have been reported to restitute a function to CFTR with specific mutations. Other molecules act on channels other than CFTR, and may be effective by bypassing CFTR itself. In the present review the various therapeutical strategies currently investigated are discussed.
自从囊性纤维化缺陷基因被克隆以来,人们对囊性纤维化跨膜传导调节因子(CFTR)的功能及其表达调控机制有了很多了解。基于目前对肺部疾病进展相关过程的认识,已经开发了一些使用基因治疗和药物制剂的方法。据报道,其中几种药物可恢复具有特定突变的CFTR的功能。其他分子作用于CFTR以外的通道,可能通过绕过CFTR本身而发挥作用。在本综述中,将讨论目前正在研究的各种治疗策略。