Gene therapy for the treatment of cystic fibrosis: A brief review

Abstract

Cystic fibrosis (CF) is a life-threatening autosomal recessive disorder resulting from mutations in the CF transmembrane conductance regulator (CFTR) gene, impacting epithelial cells of the respiratory, gastrointestinal, and reproductive systems. Although the disease has been known for a long time, there is still no effective and well-established treatment for the condition. Lung ailments, caused by pulmonary tract obstruction and secondary infections, are the major cause of morbidity and mortality in CF patients. Since the cloning of the CFTR gene in 1989, there has been significant interest in developing gene therapies for CF, with a focus on treating lung disease. Over the decades, significant advances have been made in the development of vectors for the efficient delivery of the functional CFTR gene to the pulmonary tract and in designing better pharmacologic CFTR modulators to correct malfunctioning CFTR protein. This review aims to summarize some significant breakthroughs in the development of gene therapies for CF, the challenges encountered at every stage, the lessons learned from past trials, and measures taken to address them in recent advancements, including the role of emerging technologies such as clustered regularly interspaced short palindromic repeats/Cas9 gene editing. Moreover, this review briefly discusses other promising developments in CF treatment, such as alternative therapeutic strategies, safer and more effective delivery vectors, emerging gene editing methodologies, and the development of improved animal models.

Keywords

Cystic fibrosis / Cystic fibrosis transmembrane conductance regulator / Gene therapies / Gene editing / Animal models

Acknowledgments

The first two authors express their gratitude to Institute of Child Health, Kolkata for the facility to use the Molecular Biology laboratory and the library of Haldia Institute of Technology.

Funding

None.

Conflict of interest

The authors declare that they have no competing interest.

Author contributions

Conceptualization: Pranab Roy

Visualization: Sandipan Nag

Writing - original draft: All authors

Writing - review & editing: Surupa Basu, Apurba Ghosh, Pranab Roy

Ethics approval and consent to participate

Not applicable.

Consent for publication

Not applicable.

Availability of data

All the data in this review are available in open access literature and journals.

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