Correcting genetic diseases using precision genome editing
Official title: In vivo precision genome editing to correct genetic disease
Funded at Johns Hopkins University · NIH-11052618 · Data updated 2026-11-12
This study is exploring new ways to fix genetic problems that cause heart issues like dilated cardiomyopathy, using special techniques that could allow for a one-time injection to make lasting changes, and it's currently being tested in mice to see how well it works.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Johns Hopkins University NIH-funded |
| Lab location | 1 site (Baltimore, United States) |
| Project start | April 1, 2023 |
| Project end | March 31, 2026 |
| Funding year | 2025 |
| Award (this year) | $249,000 |
| NIH project number | 5R00HL163805-04 |
| Project ID | NIH-11052618 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research include individuals diagnosed with genetic diseases, particularly those with conditions like dilated cardiomyopathy.
✕Less likely to help
Patients with genetic diseases that do not involve the types of mutations targeted by this research may not benefit.
What this research studies
This research focuses on developing innovative therapies to correct genetic mutations responsible for diseases, particularly targeting conditions like dilated cardiomyopathy. By utilizing advanced genome editing techniques, such as base and prime editors, the goal is to achieve precise corrections in the genetic material of patients through a single injection. The research employs mouse models to test the effectiveness of these editing technologies, aiming to provide a long-lasting solution to genetic disorders that currently lack approved treatments.
Why it matters
Potential benefit: If successful, this research could lead to effective therapies that permanently correct genetic defects, significantly improving the quality of life for patients with genetic diseases.
How similar studies have gone: Previous research has shown promising results with genome editing technologies, indicating potential for success in this novel approach.
Where this research is happening
-
Johns Hopkins University
Baltimore, United States Active
Researchers
- Principal investigator: Newby, Gregory a. — Johns Hopkins University
- Study coordinator: Newby, Gregory a.
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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.