Developing gene editing therapies for cystic fibrosis
Developing Gene Editing Therapeutics, Biodegradable Polymeric Delivery Vehicles, and High-throughput Platforms for the Treatment of Cystic Fibrosis
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 ID | NIH-10868613 on NIH RePORTER |
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.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with cystic fibrosis, particularly those with the W1282X mutation in the CFTR gene.
Not a fit: Patients without cystic fibrosis or those with mutations not targeted by this research may not receive any benefit.
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 performed: 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
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.