Abstract
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 language | English |
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Title of host publication | Cystic Fibrosis - Facts, Management and Advances |
Subtitle of host publication | Recent Advances in Targeted Genetic Medicines for Cystic Fibrosis |
Editors | Prashant Mohite |
Publisher | IntechOpen |
Pages | 142-163 |
Number of pages | 21 |
ISBN (Electronic) | 978-1-83881-075-7 |
ISBN (Print) | 978-1-83881-073-3 |
DOIs | |
Publication status | Published - 22 Apr 2021 |
Keywords
- 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