Correcting genetic diseases using precision genome editing
In vivo precision genome editing to correct genetic disease
['FUNDING_OTHER'] · JOHNS HOPKINS UNIVERSITY · NIH-11052618
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
| Phase | ['FUNDING_OTHER'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | JOHNS HOPKINS UNIVERSITY (nih funded) |
| Locations | 1 site (BALTIMORE, UNITED STATES) |
| Trial ID | NIH-11052618 on ClinicalTrials.gov |
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.
Who could benefit from this research
Good fit: Ideal candidates for this research include individuals diagnosed with genetic diseases, particularly those with conditions like dilated cardiomyopathy.
Not a fit: Patients with genetic diseases that do not involve the types of mutations targeted by this research may not benefit.
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 performed: Previous research has shown promising results with genome editing technologies, indicating potential for success in this novel approach.
Where this research is happening
BALTIMORE, UNITED STATES
- JOHNS HOPKINS UNIVERSITY — BALTIMORE, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: NEWBY, GREGORY A. — JOHNS HOPKINS UNIVERSITY
- Study coordinator: NEWBY, GREGORY A.
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.