Program in Developmental and Stem Cell Biology, Hospital for Sick Children, 686 Bay Street, PGCRL 16-9420, Toronto, ON, M5G0A4, Canada.
Department of Laboratory Medicine and Pathobiology, University of Toronto, Toronto, Canada.
J Transl Med. 2021 Oct 30;19(1):452. doi: 10.1186/s12967-021-03099-4.
The discovery of the Cystic fibrosis (CF) gene in 1989 has paved the way for incredible progress in treating the disease such that the mean survival age of individuals living with CF is now ~58 years in Canada. Recent developments in gene targeting tools and new cell and animal models have re-ignited the search for a permanent genetic cure for all CF. In this review, we highlight some of the more recent gene therapy approaches as well as new models that will provide insight into personalized therapies for CF.
1989 年囊性纤维化（CF）基因的发现为治疗该病带来了令人难以置信的进展，使得现在加拿大 CF 患者的平均存活年龄约为 58 岁。基因靶向工具的最新发展以及新型细胞和动物模型重新点燃了寻找所有 CF 永久基因治疗方法的研究热情。在这篇综述中，我们重点介绍了一些最近的基因治疗方法以及新的模型，这些将为 CF 的个性化治疗提供新的见解。
