Systematic Review of Emerging Technologies in Cystic Fibrosis Treatment: Gene Therapy and CRISPR Strategies for the Future

Cystic Fibrosis Treatment

Abstract

The current systematic review aimed to evaluate research on cystic fibrosis (CF), a genetic disease that affects multiple organs, particularly the lungs, and is associated with high morbidity and mortality. A total of 7,831 relevant studies were identified from search databases, and 27 studies were ultimately considered appropriate for review after applying eligibility criteria, including three longitudinal studies and the remainder being cross-sectional. All studies included a healthy control group, with a combined total of 1,839 individuals with CF and 2,178 controls. The age range varied across studies; however, the majority were conducted in adults.The studies had different aims, including evaluating and comparing different techniques for gene therapy and CRISPR, and assessing changes in body nutrition status. Other studies focused on the evaluation of lung function, inflammation, and clinical parameters. Animal models have played a crucial role in advancing CF gene therapy. Various animal models have been developed, including pigs, ferrets, rats, zebrafish, and sheep, each with its advantages and limitations. The CF pig model has facilitated the measurement of CFTR correction in vivo and has helped define the relationship between CFTR expression and Cl– and HCO3– transport, with important implications for CF gene therapies. Gene editing technologies, such as CRISPR/Cas9, have emerged as promising approaches to modifying nucleic acid sequences in CF research. These tools hold the potential to repair the endogenous CFTR gene and restore its function, but efficient in vivo gene delivery remains a significant challenge. Assessing changes in body composition can provide valuable information on the effects of gene therapy or CRISPR on the overall health of CF patients. The assessment of body composition changes in CF treatment is essential, as current therapies such as CFTR modulators primarily target the respiratory system and may not fully address the systemic effects of the disease. Gene therapy and CRISPR have the potential to provide more comprehensive and long lasting treatments for CF, and assessment of body composition changes can serve as a clinical endpoint in future clinical trials.