Genetic medicines for CF: Hype versus reality

摘要

Summary Since identification of theCFTR gene over 25 years ago, gene therapy for cystic fibrosis (CF) has been actively developed. More recently gene therapy has been joined by other forms of genetic medicines including mRNA delivery, as well as genome editing and mRNA repairbased strategies. Proofofconcept that gene therapy can stabilize the progression of CF lung disease has recently been established in a Phase IIb trial. An early phase study to assess the safety and explore efficacy of CFTR mRNA repair is ongoing, while mRNA delivery and genome editingbased strategies are currently at the preclinical phase of development. This review has been written jointly by some of those involved in the various CF genetic medicine fields and will summarize the current stateoftheart, as well as discuss future developments. Where applicable, it highlights common problems faced by each of the strategies, and also tries to highlight where a specific strategy may have an advantage on the pathway to clinical translation. We hope that this review will contribute to the ongoing discussion about the hype versus reality of genetic medicinebased treatment approaches in CF.Pediatr Pulmonol. 2016;51:S5S17. ?2016 Wiley Periodicals, Inc.