Developing gene editing therapies for cystic fibrosis
Official title: Developing Gene Editing Therapeutics, Biodegradable Polymeric Delivery Vehicles, and High-throughput Platforms for the Treatment of Cystic Fibrosis
Funded at University of Michigan at Ann Arbor · NIH-10868613 · Data updated 2026-11-12
This study is exploring new gene editing treatments for cystic fibrosis, especially for those with a specific mutation, to help fix the genetic problem at its source and potentially offer a cure.
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 start | July 1, 2023 |
| Project end | June 30, 2026 |
| Funding year | 2024 |
| Award (this year) | $248,999 |
| NIH project number | 5R00HL151806-04 |
| Project ID | NIH-10868613 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with cystic fibrosis, particularly those with the W1282X mutation in the CFTR gene.
✕Less likely to help
Patients without cystic fibrosis or those with mutations not targeted by this research may not receive any benefit.
What this research studies
This research focuses on creating innovative gene editing therapies to treat cystic fibrosis, particularly targeting the severe W1282X mutation in the CFTR gene. The approach utilizes advanced techniques like CRISPR/Cas9 and prime editing to correct genetic mutations, while also developing biodegradable delivery systems to ensure effective treatment delivery to patients. By employing high-throughput platforms, the research aims to identify the best methods for delivering these therapies safely and effectively within the body. Patients may benefit from a potential curative treatment that addresses the underlying genetic cause of their condition.
Why it matters
Potential benefit: If successful, this research could provide a curative treatment option for cystic fibrosis patients with specific genetic mutations.
How similar studies have gone: Previous research using CRISPR and prime editing technologies has shown promise in correcting genetic mutations, indicating potential success for this approach.
Where this research is happening
-
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
Related research
Other NIH-funded projects on the same topics.
-
Blocking CFTR in the cervix to prevent conception without hormones
A non-hormonal approach aims to stop sperm from entering the uterus by making cervical mucus too thick for sperm, potentially offering a new contraception option.
-
Optimizing Treatments for People with Cystic Fibrosis on Highly Effective Modulator Therapy
A real-world study in people with cystic fibrosis on highly effective CFTR modulator therapy to understand how traditional CF treatments can be adjusted safely.
-
Elastin-like particles to deliver gene therapies for cystic fibrosis
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.
-
Pharmacogenomic Predictors of Clinical Responses in Cystic Fibrosis using Targeted WGS
A study looking at how genetic information from whole-genome sequencing can predict how people with cystic fibrosis respond to medications, using a focused set of pharmacogenomic genes.
-
Ceftaroline-meropenem combo to beat MRSA in cystic fibrosis
A new combination antibiotic approach is being tested to help people with cystic fibrosis who have MRSA infections that resist standard treatments.
-
Gut microbiome in cystic fibrosis and colorectal cancer risk
Researchers are studying how the gut bacteria in adults with cystic fibrosis influence the development of colorectal cancer.
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.