New approaches to genetic therapies for cystic fibrosis

A. Christopher Boyd, Shuling Guo, Lulu Huang, Batsheva Kerem, Yifat S. Oren, Amy J. Walker, Stephen L. Hart*

*Corresponding author for this work

Research output: Contribution to journalReview articlepeer-review

44 Scopus citations

Abstract

Gene therapy offers great promise for cystic fibrosis which has never been quite fulfilled due to the challenges of delivering sufficient amounts of the CFTR gene and expression persistence for a sufficient period of time in the lungs to have any effect. Initial trials explored both viral and non-viral vectors but failed to achieve a significant breakthrough. However, in recent years, new opportunities have emerged that exploit our increased knowledge and understanding of the biology of CF and the airway epithelium. New technologies include new viral and non-viral vector approaches to delivery, but also alternative nucleic acid technologies including oligonucleotides and siRNA approaches for gene silencing and gene splicing, described in this review, as presented at the 2019 annual European CF Society Basic Science meeting (Dubrovnik, Croatia). We also briefly discuss other emerging technologies including mRNA and CRISPR gene editing that are advancing rapidly. The future prospects for genetic therapies for CF are now diverse and more promising probably than any time since the discovery of the CF gene.

Original languageAmerican English
Pages (from-to)S54-S59
JournalJournal of Cystic Fibrosis
Volume19
DOIs
StatePublished - Mar 2020

Bibliographical note

Publisher Copyright:
© 2019

Keywords

  • Antisense oligonucleotides
  • CFTR
  • Cystic fibrosis
  • ENaC
  • Gene therapy
  • Liposomes
  • Nanoparticles
  • mRNA splicing
  • siRNA

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