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囊状纤维化基因的克隆成功,使得本病的基因治疗成为可能。本文综述基因治疗囊状纤维化的理论基础和初步结果,对几种具有临床应用价值的载体和安全性作一重点介绍。
The successful cloning of cystic fibrosis gene makes the gene therapy of this disease possible. This review summarizes the theoretical basis and preliminary results of gene therapy for cystic fibrosis, and gives a brief introduction of several vectors and safety with clinical application value.