Using precision editing to treat motor neuron diseases
Precision Base Editing for the Treatment of Motor Neuron Diseases
This study is exploring a new way to treat motor neuron diseases like spinal muscular atrophy and ALS by using cutting-edge gene editing techniques to fix the genetic problems that cause these conditions, with the hope of creating personalized treatments that work better for each person.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Boston Children's Hospital NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project ID | NIH-10928811 on NIH RePORTER |
What this research studies
This research focuses on developing a new therapeutic approach for motor neuron diseases (MNDs) like spinal muscular atrophy (SMA) and amyotrophic lateral sclerosis (ALS) by utilizing advanced genome editing techniques. The goal is to create a general strategy that can effectively target and correct genetic mutations responsible for these diseases. By understanding the factors that influence the efficiency and precision of base editing, the researchers aim to design tailored treatments that can address the unique genetic abnormalities in each patient. This innovative approach could potentially lead to more effective therapies for individuals suffering from these debilitating conditions.
Who could benefit from this research
Good fit: Ideal candidates for this research are adults over 21 years old who have been diagnosed with motor neuron diseases such as ALS or SMA.
Not a fit: Patients with motor neuron diseases caused by genetic mutations not targeted by this research may not receive any benefit.
Why it matters
Potential benefit: If successful, this research could provide targeted genetic therapies that significantly improve outcomes for patients with motor neuron diseases.
How similar studies have performed: Other research has shown promise in using genome editing techniques for genetic disorders, indicating potential success for this novel approach.
Where this research is happening
Boston, United States
- Boston Children's Hospital — Boston, United States (Active)
Researchers
- Principal investigator: Arbab, Mandana — Boston Children's Hospital
- Study coordinator: Arbab, Mandana
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.