Colledge W H
Wellcome/CRC Institute of Cancer and Developmental Biology, Cambridge, UK.
Curr Opin Genet Dev. 1994 Jun;4(3):466-71. doi: 10.1016/0959-437x(94)90037-x.
A variety of cystic fibrosis gene therapy approaches based on viral (adenovirus, retrovirus, and adeno-associated virus) and non-viral (liposomes and receptor-mediated endocytosis) routes are currently being assessed for safety and efficacy. Of these, the trials involving liposomal and adenoviral vectors are the most advanced, as both have been shown to correct the cystic fibrosis Cl- conductance defect in vivo.
目前正在评估多种基于病毒（腺病毒、逆转录病毒和腺相关病毒）和非病毒（脂质体和受体介导的内吞作用）途径的囊性纤维化基因治疗方法的安全性和有效性。其中，涉及脂质体和腺病毒载体的试验最为先进，因为两者都已被证明能在体内纠正囊性纤维化氯离子传导缺陷。
