Exploring Gene Therapy Strategies for Cystic Fibrosis: A Comprehensive Review of CFTR Gene Mutations, Bioinformatics Analysis, and Emerging Therapeutic Approaches

Aim: This study aimed to identify the most promising gene therapy approach for treating patients with cystic fibrosis.

Background: Cystic fibrosis is a rare autosomal recessive disorder affecting malabsorption, malnutrition, and lung function. It results from genetic mutations in the CFTR gene, responsible for producing the CFTR protein, a channel for chloride ions. Existing research highlights lentiviral and adenoviral vectors as highly effective for gene therapy in cystic fibrosis patients.

Methods: The NCBI database, specifically GenBank, was utilized to extract the DNA sequence of the CFTR gene, including its accession number, amino acid count, and number of exons. FASTA format facilitated the retrieval of nucleotide sequences and the assessment of protein function. BLAST was employed to compare CFTR gene protein products between humans and Mus musculus (house mice). Gene therapy interventions were then applied to the animal model to ensure safety and efficacy.

Results: The CFTR gene's accession number is NC_000007.14, with a protein product comprising 1480 amino acids and 27 exons. The gene is located on chromosome 7. Importantly, the house mouse was found to possess the same gene as humans.

Conclusion: Utilizing adenoviral and lentiviral vectors emerges as a safe and effective gene therapy approach, mitigating complications associated with cystic fibrosis.