Emerging technologies for cystic fibrosis transmembrane conductance regulator restoration in all people with CF

被引:9
作者
Egan, Marie E. [1 ]
机构
[1] Yale Univ, Sch Med, Dept Pediat, Div Pulm Allergy Immunol Sleep Med, New Haven, CT 06510 USA
关键词
cystic fibrosis; DNA/RNA technologies; GENE-THERAPY; DOUBLE-BLIND; TEZACAFTOR-IVACAFTOR; ADENOVIRUS VECTOR; CLINICAL-TRIAL; STEM-CELLS; MUTATION; SAFETY; REPAIR; EPITHELIA;
D O I
10.1002/ppul.24965
中图分类号
R72 [儿科学];
学科分类号
100202 ;
摘要
Although effective cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapy has the potential to change the lives of many patients with cystic fibrosis (CF), it is unlikely that these drugs will be a game changing therapy for all. There are about 10% of patients with CF who don't produce a mutant protein tomodulate, potentiate, or optimize and for these patients such therapies are unlikely to be of significant benefit. There is a need to develop new therapeutic approaches that can work for this patient population and can advance CF therapies. These new therapies will be genetic-based therapies and each approach will result in functional CFTR protein inpreviously affected CF cells. In this review we will examine the potential of RNA therapies, gene transfer therapies, and gene editing therapies for the treatment of CF as well as the challenges that will need to be facedas we harness the power of these emerging therapies towards a one-time cure.
引用
收藏
页码:S32 / S39
页数:8
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