Elastin-like particles to deliver gene therapies for cystic fibrosis
Official title: Elastin-Like Polypeptide Nanoparticles for Precision Gene Therapy of Cystic Fibrosis
Funded at Beth Israel Deaconess Medical Center · NIH-11447142 · Data updated 2026-11-12
A new nanoparticle system is being developed to deliver genetic therapies directly to the lungs of people with cystic fibrosis, including those with nonsense mutations.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Beth Israel Deaconess Medical Center NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project start | September 1, 2026 |
| Project end | May 31, 2030 |
| Funding year | 2026 |
| Award (this year) | $852,908 |
| NIH project number | 1R01HL183196-01A1 |
| Project ID | NIH-11447142 on NIH RePORTER |
Who could benefit from this research
✓Could help
People with cystic fibrosis, especially those with nonsense CFTR mutations, who could be candidates for future gene or genome-editing therapies.
✕Less likely to help
Patients without access to upcoming gene-delivery therapies or those for whom inhaled gene therapies are unsuitable may not benefit.
What this research studies
Researchers are refining elastin-like polypeptide nanoparticles that can carry genetic material or genome-editing tools into lung cells. The project combines design, computer modeling, and lab testing to create particles that release their cargo in response to lung cell conditions. If successful, this could enable precise, lung-targeted treatments that bypass current modulators and address mutations not benefiting from existing therapies.
Why it matters
Potential benefit: If successful, patients could gain more effective, personalized gene-based treatments for cystic fibrosis.
How similar studies have gone: Similar nanoparticle approaches have shown promise in delivering genetic therapies in preclinical models, with some systems achieving efficient lung delivery in animals.
Where this research is happening
-
Beth Israel Deaconess Medical Center
Boston, United States Active
Researchers
- Principal investigator: Chaikof, Elliot — Beth Israel Deaconess Medical Center
- Study coordinator: Chaikof, Elliot
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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.