Gene Editing for Cystic Fibrosis
Developing Gene Editing Therapeutics, Biodegradable Polymeric Delivery Vehicles, and High-throughput Platforms for the Treatment of Cystic Fibrosis
This research explores new ways to fix the genetic cause of cystic fibrosis using advanced gene editing tools.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Michigan at Ann Arbor NIH-funded |
| Lab location | 1 site (Ann Arbor, United States) |
| Project ID | NIH-11121060 on NIH RePORTER |
What this research studies
Our goal is to create a lasting treatment for cystic fibrosis, especially for patients with severe mutations like W1282X. We are working with gene editing technologies, including CRISPR/Cas9 prime editing and triplex-forming peptide nucleic acids (PNAs), to correct the faulty gene. We are also designing special biodegradable carriers to safely deliver these gene editing tools to the right cells in the body. This work aims to overcome current challenges in getting gene therapies to where they need to go effectively.
Who could benefit from this research
Good fit: This research is focused on patients with cystic fibrosis, particularly those with specific genetic mutations like W1282X.
Not a fit: Patients without cystic fibrosis or those with different genetic conditions would not directly benefit from this specific gene editing approach.
Why it matters
Potential benefit: If successful, this research could lead to a curative gene editing treatment for cystic fibrosis, offering a permanent solution for patients.
How similar studies have performed: Other gene editing systems like CRISPR/Cas9 have shown promise and are already being tested in clinical trials for various conditions.
Where this research is happening
Ann Arbor, United States
- University of Michigan at Ann Arbor — Ann Arbor, United States (Active)
Researchers
- Principal investigator: Piotrowski-Daspit, Alexandra Sarah Annukka — University of Michigan at Ann Arbor
- Study coordinator: Piotrowski-Daspit, Alexandra Sarah Annukka
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 below.