Lallemand Jean-Yves, Leclaire Bayesté, Sermet Isabelle, Lenoir Gérard
ICSN-RMN, Ecole Polytechnique, 91128 Palaiseau.
Bull Acad Natl Med. 2005 May;189(5):789-95; discussion 795-6.
Cystic Fibrosis is the most common lethal genetic disease among Caucasian population. Despite considerable efforts, no significant progress has been so far achieved by gene therapies approaches. On the basis of a surprising clinical observation, we have developed an approach using anti-cancer drugs promoting the over expression of ABC transporters closely related to the deficient protein CFTR, which seem able to share functions with it and to restore the missing function(s).
囊性纤维化是白种人群中最常见的致死性遗传疾病。尽管付出了巨大努力，但迄今为止基因治疗方法尚未取得显著进展。基于一项惊人的临床观察，我们开发了一种方法，即使用抗癌药物促进与缺陷蛋白CFTR密切相关的ABC转运蛋白的过度表达，这些转运蛋白似乎能够与CFTR共享功能并恢复缺失的功能。
