Creating new tools to edit RNA for treating genetic diseases
Official title: Developing programmable RNA writing tools with the novel RNA-guided RNA-targeting CRISPR effector Cas7-11
Funded at Beth Israel Deaconess Medical Center · NIH-11141415 · Data updated 2026-11-12
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
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Beth Israel Deaconess Medical Center NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project start | September 23, 2023 |
| Project end | August 31, 2027 |
| Funding year | 2024 |
| Award (this year) | $460,261 |
| NIH project number | 7R01GM148745-02 |
| Project ID | NIH-11141415 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals with genetic disorders, especially those characterized by triplet repeat expansions.
✕Less likely to help
Patients with conditions that do not involve RNA modifications or triplet repeat disorders may not benefit from this research.
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.
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 gone: 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
-
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
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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.