Systematic Review of Emerging Technologies in Cystic Fibrosis Treatment: Gene Therapy and CRISPR Strategies for the Future
Keywords:
Cystic Fibrosis, genetic disease, systematic review, morbidity, mortality, gene therapy, CRISPR/Cas9, CFTR gene, CFTR modulators, systemic effects, clinical trials, nucleic acid modification, endogenous CFTR repair, health assessment, respiratory system.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 7831 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 remaining cross-sectional ones. All studies included a healthy control group, with a combined total of 1839 individuals with CF and 2178 controls. The age range varied across studies, but the majority were carried out 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, which enhances the correlation of CFTR expression and transportation of Cl- and HCO3 as a result decreases the implication of cystic fibrosis in gene therapy. 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 proper evaluation of nutritional composition in the 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. The CRISPR techniques and gene therapy treatment have the potential to provide more comprehensive and long-lasting treatments for CF and the assessment of body composition changes is essential for future clinical trials.
