Personalized therapeutic oligonucleotides for primary ciliary dyskinesia
Official title: Targeting Primary Ciliary Dyskinesia with Personalized Therapeutic Oligonucleotides
Funded at Univ of North Carolina Chapel Hill · NIH-11432843 · Data updated 2026-11-12
A new therapy approach uses customized RNA-based medicines to help people with Primary Ciliary Dyskinesia, a rare lung condition, by correcting how genes are read in airway cells.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Univ of North Carolina Chapel Hill NIH-funded |
| Lab location | 1 site (Chapel Hill, United States) |
| Project start | September 2, 2026 |
| Project end | May 31, 2030 |
| Funding year | 2026 |
| Award (this year) | $563,936 |
| NIH project number | 1R01HL188024-01 |
| Project ID | NIH-11432843 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are individuals with PCD caused by specific splicing mutations (e.g., CCDC39/CCDC40) or similar genotypes where SSOs could correct splicing.
✕Less likely to help
Patients without the targeted splicing mutations or with non-amenable genetic alterations may not benefit.
What this research studies
This project aims to develop splice-switching oligonucleotides tailored to individual genetic mutations causing PCD, with a focus on restoring proper ciliary function in airway tissues. It builds on lab data showing restored splicing and improved mucociliary transport in patient-derived nasal cells. The work will progress from cellular models toward patient-relevant applications, considering personalized treatment options for those with specific mutations. The study emphasizes a platform approach that could support N-of-1 therapies for orphan lung diseases, with careful safety and efficacy evaluation in relevant models.
Why it matters
Potential benefit: If successful, patients with PCD could receive personalized RNA medicines that improve airway clearance and reduce infections.
How similar studies have gone: Preliminary lab results show restored function in patient-derived cells, supporting potential translation to patients with similar genetic defects.
Where this research is happening
-
Univ of North Carolina Chapel Hill
Chapel Hill, United States Active
Researchers
- Principal investigator: Kreda, Silvia Marina — Univ of North Carolina Chapel Hill
- Study coordinator: Kreda, Silvia Marina
Related research
Other NIH-funded projects on the same topics.
-
Pathogenesis and New Treatments for Primary Ciliary Dyskinesia (PCD)
Researchers are studying why Primary Ciliary Dyskinesia causes lung and airway problems and how to develop better treatments for people with this condition.
-
New genes behind primary ciliary dyskinesia
This project looks for new genes that cause primary ciliary dyskinesia to help people with chronic respiratory infections and related complications.
-
How a lung cell protein (cytoglobin) affects cilia movement and organ placement
Researchers are looking at whether the airway protein cytoglobin controls nitric oxide and cilia motion in ways that could explain primary ciliary dyskinesia and situs inversus.
-
Understanding and treating Primary Ciliary Dyskinesia
This study is looking at a pig model of Primary Ciliary Dyskinesia (PCD) to better understand how the condition affects breathing and to find new ways to help improve the lives of people living with PCD.
-
Finding new genes that cause primary ciliary dyskinesia
This study is looking for new genes that cause primary ciliary dyskinesia (PCD), a condition that affects breathing, to help make faster and cheaper tests for it, using a special model from frog embryos to learn more about how cilia work.
-
Understanding how a protein affects cilia movement and organ development in airway diseases.
This study is looking at how a protein called cytoglobin helps tiny hair-like structures in your airways work properly, which is important for people with primary ciliary dyskinesia (PCD) and related breathing problems, with the hope of finding better ways to diagnose and treat these conditions.
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page.