Investigating a new CRISPR method for treating genetic causes of Alzheimer's disease
Genetic foundation for complete mutant allele-specific CRISPR in neurodegenerative diseases
This study is exploring a new way to use gene editing to target the specific genetic changes that cause neurodegenerative diseases like Alzheimer's, which could lead to better treatments that are customized for each patient's unique genetics.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Massachusetts General Hospital NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project ID | NIH-10447597 on NIH RePORTER |
What this research studies
This research focuses on developing a novel CRISPR gene editing technique that targets specific genetic mutations responsible for neurodegenerative diseases like Alzheimer's. By using a method that alters PAM sites on mutant alleles, the approach aims to silence disease-causing genes with high precision. This could potentially lead to more effective treatments by directly addressing the genetic roots of these conditions. Patients may benefit from therapies that are tailored to their specific genetic profiles, improving treatment outcomes.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with genetic forms of Alzheimer's disease or those carrying specific mutations linked to the condition.
Not a fit: Patients with non-genetic forms of Alzheimer's disease or those without the specific mutations targeted by this research may not benefit.
Why it matters
Potential benefit: If successful, this research could lead to more effective and targeted therapies for patients with genetic forms of Alzheimer's disease.
How similar studies have performed: Other research has shown promise in using CRISPR technology for gene editing, indicating potential success for this novel approach.
Where this research is happening
Boston, United States
- Massachusetts General Hospital — Boston, United States (Active)
Researchers
- Principal investigator: Lee, Jong-Min — Massachusetts General Hospital
- Study coordinator: Lee, Jong-Min
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.