Improving gene editing techniques for treating neurological diseases
Official title: Advanced Delivery Platforms for Base Editing In Vivo
Funded at Univ of Massachusetts Med Sch Worcester · NIH-10912556 · Data updated 2026-11-12
This study is testing a new way to improve gene editing for brain and nervous system conditions using a special tool called Nme2Cas9, which helps make precise changes in genes while aiming to keep side effects low and treatment safe.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Univ of Massachusetts Med Sch Worcester NIH-funded |
| Lab location | 1 site (Worcester, United States) |
| Project start | September 1, 2023 |
| Project end | August 31, 2027 |
| Funding year | 2024 |
| Award (this year) | $583,738 |
| NIH project number | 5R01GM150273-02 |
| Project ID | NIH-10912556 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals with genetic disorders affecting the central nervous system, such as Amyotrophic Lateral Sclerosis or Batten Disease.
✕Less likely to help
Patients with conditions unrelated to genetic mutations or those not affecting the central nervous system may not benefit from this research.
What this research studies
This research focuses on enhancing gene editing methods using a novel platform called Nme2Cas9, which allows for precise alterations in the genome. By utilizing adeno-associated viruses (AAV) for delivery, the project aims to improve the efficiency and safety of base editing in vivo, particularly for conditions affecting the central nervous system. The researchers will validate these advanced editing systems in mouse models of CNS diseases, aiming to minimize side effects and enhance therapeutic outcomes.
Why it matters
Potential benefit: If successful, this research could lead to more effective and safer treatments for neurological diseases caused by genetic mutations.
How similar studies have gone: Previous research has shown promise in using CRISPR-based approaches for gene editing, indicating potential success for this novel method.
Where this research is happening
-
Univ of Massachusetts Med Sch Worcester
Worcester, United States Active
Researchers
- Principal investigator: Sontheimer, Erik J. — Univ of Massachusetts Med Sch Worcester
- Study coordinator: Sontheimer, Erik J.
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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.