Developing a new CRISPR method to treat genetic muscle disorders.
Official title: Translational development of novel CRISPR approach to treat genomic duplications
Funded at Myofinity Biosciences INC · NIH-10698879 · Data updated 2026-11-12
This study is testing a new CRISPR treatment that aims to fix the genetic changes causing Duchenne muscular dystrophy by removing extra DNA copies, which could help restore the healthy protein needed for muscle function, and it's being tested in mice that have a similar condition to humans.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Myofinity Biosciences INC NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project start | August 1, 2023 |
| Project end | July 31, 2025 |
| Funding year | 2023 |
| Award (this year) | $275,651 |
| NIH project number | 1R41AR082205-01A1 |
| Project ID | NIH-10698879 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with Duchenne muscular dystrophy who have specific duplication mutations in their dystrophin gene.
✕Less likely to help
Patients with Duchenne muscular dystrophy caused by mutations other than duplications may not benefit from this research.
What this research studies
This research focuses on creating a novel CRISPR-based treatment aimed at correcting genetic duplications that cause Duchenne muscular dystrophy (DMD). By utilizing a specific SaCas9 nickase system, the project seeks to restore the production of healthy dystrophin protein, which is crucial for muscle function. The approach involves removing duplicated sections of DNA to restore the normal gene sequence, potentially offering a targeted therapy for patients with specific genetic mutations. The research will be conducted using a mouse model that mimics human DMD, allowing for effective testing of the treatment's efficacy.
Why it matters
Potential benefit: If successful, this research could provide a targeted therapy for patients with Duchenne muscular dystrophy, improving muscle function and quality of life.
How similar studies have gone: Previous research has shown promise in using CRISPR technology for genetic corrections, indicating potential success for this novel approach.
Where this research is happening
-
Myofinity Biosciences INC
Boston, United States Active
Researchers
- Principal investigator: Lek, Monkol — Myofinity Biosciences INC
- Study coordinator: Lek, Monkol
Related research
Other NIH-funded projects on the same topics.
-
Membrane-repair protein treatment for Becker muscular dystrophy
This project is developing a modified protein medicine to help muscle cells patch their damaged outer membrane in people with Becker muscular dystrophy.
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Improving muscle repair for Becker Muscular Dystrophy
This study is testing a new protein treatment designed to help repair muscle cells for people with Becker Muscular Dystrophy, with the hope of improving muscle function and easing symptoms.
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.