Recent Advances in Targeted Genetic Medicines for Cystic Fibrosis

Research output: Chapter in Book/Report/Conference proceedingChapterpeer-review


The cystic fibrosis transmembrane conductance regulator (CFTR) gene was discovered just over 30 years ago, and soon after, gene therapy for cystic fibrosis (CF) has been rapidly and continually developing. Recently, novel gene therapy strategies have been developed, including mRNA delivery, genome editing, and mRNA repair; all these strategies are collectively named “genetic medicines.” The last quarter of the century showed a significant boost in the development of viral and nonviral vectors to deliver genetic treatment. This chapter will provide a brief overview of the CFTR gene and its different classes of mutations as well as a review of the different genetic therapeutic options that are under research. Later in this chapter, drugs that target different CFTR mutation classes and are currently approved to treat CF patients will be briefly presented.
Original languageEnglish
Title of host publicationCystic Fibrosis - Facts, Management and Advances
Subtitle of host publicationRecent Advances in Targeted Genetic Medicines for Cystic Fibrosis
EditorsPrashant Mohite
Number of pages21
ISBN (Electronic)978-1-83881-075-7
ISBN (Print)978-1-83881-073-3
Publication statusPublished - 22 Apr 2021


  • cystic fibrosis
  • CFTR
  • gene therapy
  • CRISPR/Casg
  • mRNA therapy
  • gene editing
  • gene delivery
  • viral vectors
  • nonviral vectors
  • CF animal models
  • CF drugs

Research Institutes

  • Health Research Institute

Research Centres

  • Cardio-Respiratory Research Centre


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