Gene Therapy for Ciliary Beating Recovery in Airway Cells
Author Information
Author(s): Chhin Brigitte, Negre Didier, Merrot Olivier, Pham Jacqueline, Tourneur Yves, Ressnikoff Denis, Jaspers Martine, Jorissen Mark, Cosset François-Loïc, Bouvagnet Patrice
Primary Institution: Université de Lyon, Lyon, France
Hypothesis
Gene therapy could restore ciliary function in DNAI1-mutated airway epithelial cells to prevent patients from infectious complications.
Conclusion
The study demonstrated that gene therapy can restore ciliary beating in DNAI1-deficient human airway epithelial cells.
Supporting Evidence
- Transduction of DNAI1-mutated HAECs with wild-type DNAI1 can restore ciliary beating.
- Normal ciliary beating was recorded in treated cells, while untreated cells remained immotile.
- Electron microscopy showed the presence of outer dynein arms in treated cells.
Takeaway
Scientists used a special virus to fix the tiny hairs in the lungs of a sick person, helping them to work properly again.
Methodology
Lentiviral vectors were used to transduce DNAI1-deficient human airway epithelial cells, and ciliary beating was assessed post-treatment.
Limitations
The study was conducted on a single patient, and the transduction efficiency was not optimal.
Participant Demographics
A male patient with Primary Ciliary Dyskinesia and compound heterozygous mutations in the DNAI1 gene.
Statistical Information
P-Value
p<0.0001
Statistical Significance
p<0.0001
Digital Object Identifier (DOI)
Want to read the original?
Access the complete publication on the publisher's website