Gene Editing for Cystic Fibrosis: Advances and Prospects of CRISPR‐Cas9 Therapy

Sep 22, 2025Cell biology international

Gene Editing Using CRISPR-Cas9 for Treating Cystic Fibrosis: Progress and Future Possibilities

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Abstract

Genome editing technologies may offer a permanent treatment for cystic fibrosis by correcting genetic mutations.

Cystic fibrosis is caused by mutations in the CFTR gene, which is essential for maintaining water and salt balance in the body.
Impairment of CFTR affects multiple organs, particularly the lungs.
Current treatments, such as CFTR modulators, target specific mutations but have limited therapeutic potential.
Genome editing technologies like CRISPR-Cas9 could potentially correct various CFTR mutations.
The review discusses the development and prognosis of cystic fibrosis, alongside advancements in gene editing for treatment.

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Cystic fibrosis (CF) is an inherited, autosomal recessive disorder that is caused by mutations in the gene encoding cystic fibrosis transmembrane conductance regulator (CFTR). CFTR maintains the balance between water and salts by transporting chloride ions along various epithelial surfaces. CFTR impairment affects the function of several organs, including the lungs. Newborn screening, prenatal diagnosis, and pharmacological interventions have altered the prevalence and incidence of cystic fibrosis. Although CFTR modulators are a promising treatment option, their ability to target and correct only one mutation at a time restricts their therapeutic potential. The development of genome editing technologies such as Clustered Regularly Interspaced Short Palindromic Repeats-Cas(CRISPR-Cas9) has the potential to correct genetic mutations, including those associated with CF, thereby offering a permanent treatment by fixing the root cause of CF. This article summarizes cystic fibrosis development, prognosis, and diagnosis, as well as possibilities for correcting various types of CFTR gene mutations. The review focuses on the potential of gene editing technologies to repair CFTR mutations and their applications in the advancement of CF treatment.

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Gene Editing for Cystic Fibrosis: Advances and Prospects of CRISPR‐Cas9 Therapy

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You found one interesting study. We’ll send the next 7.

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Recent issues from the crispr gene editing brief