期刊
JOURNAL OF TRANSLATIONAL MEDICINE
卷 19, 期 1, 页码 -出版社
BMC
DOI: 10.1186/s12967-021-03099-4
关键词
Cystic fibrosis; Stem cells; Lung; Organoids; CFTR; Alternative chloride channels; TMEM16A; Precision medicine
资金
- SickKids Foundation-CIHR IHDCYH [NI20-1070]
- Medicine by Design (University of Toronto) grants
- University of Toronto
The discovery of the Cystic fibrosis (CF) gene in 1989 has led to significant progress in treating the disease, with the average survival age of patients with CF reaching 58 years in Canada. Recent advancements in gene therapy approaches and new research models are rekindling the search for a permanent genetic cure for all CF.
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 similar to 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.
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