Creating new tools to edit RNA for treating genetic diseases
Developing programmable RNA writing tools with the novel RNA-guided RNA-targeting CRISPR effector Cas7-11
['FUNDING_R01'] · BETH ISRAEL DEACONESS MEDICAL CENTER · NIH-11141415
This study is exploring a new way to fix genetic disorders by using a special tool that edits RNA instead of DNA, which could offer a safer and more precise treatment option for patients with conditions caused by repeated genetic sequences.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | BETH ISRAEL DEACONESS MEDICAL CENTER (nih funded) |
| Locations | 1 site (BOSTON, UNITED STATES) |
| Trial ID | NIH-11141415 on ClinicalTrials.gov |
What this research studies
This research focuses on developing innovative RNA editing tools using a novel CRISPR effector called Cas7-11. The approach aims to address challenges associated with traditional DNA editing, particularly for genetic disorders that involve triplet repeats. By utilizing RNA modifications, the research seeks to provide a safer and more effective method for correcting genetic diseases, allowing for temporary and reversible changes without the risks of permanent off-target effects. Patients may benefit from a more precise treatment option that can target specific genetic issues without altering the DNA permanently.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals with genetic disorders, especially those characterized by triplet repeat expansions.
Not a fit: Patients with conditions that do not involve RNA modifications or triplet repeat disorders may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to safer and more effective treatments for genetic disorders, particularly those involving triplet repeat expansions.
How similar studies have performed: While gene editing has shown promise, this RNA editing approach is relatively novel and aims to overcome limitations seen in previous studies.
Where this research is happening
BOSTON, UNITED STATES
- BETH ISRAEL DEACONESS MEDICAL CENTER — BOSTON, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: GOOTENBERG, JONATHAN SAMUEL — BETH ISRAEL DEACONESS MEDICAL CENTER
- Study coordinator: GOOTENBERG, JONATHAN SAMUEL
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.