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Updated: Jul 25, 2025
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Generation of Human Nasal Epithelial Cell Spheroids for Individualized Cystic Fibrosis Transmembrane Conductance Regulator Study
Published on: April 11, 2018
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Genome Editing for Cystic Fibrosis
1Department of Microbiology, Immunology and Parasitology, Louisiana State University Health Sciences Center, CSRB 607, 533 Bolivar Street, New Orleans, LA 70112, USA.
Cells
|June 28, 2023
Summary
Cystic fibrosis (CF) gene therapy using CRISPR/Cas editing offers a potential permanent cure for all patients. This approach aims to repair genetic defects, addressing limitations of current CFTR modulator therapies.
Area of Science:
- Genetics
- Molecular Biology
- Biotechnology
Background:
- Cystic fibrosis (CF) is a genetic disorder caused by CFTR gene mutations.
- Current CFTR modulators treat ~90% of patients but are not curative.
- A durable, curative therapy for all CF patients is still needed.
Purpose of the Study:
- To review the progress of gene editing technologies for CF therapy.
- To discuss the potential of CRISPR/Cas gene editing for CF.
Main Methods:
- Review of recent advancements in CRISPR/Cas gene editing technology.
- Analysis of the application of gene editing for CFTR gene repair.
Main Results:
- CRISPR/Cas gene editing presents a promising platform for genetic therapy.
- This technology offers a potential route for in-situ repair of CF-causing mutations.
Conclusions:
- Gene editing technologies are rapidly evolving for CF treatment.
- CRISPR/Cas gene editing holds potential for a definitive cure for all CF patients.
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