New genetic clues and cells to understand ALS
Official title: Discovery and functional analysis of new ALS genetic drivers
Funded at Univ of Massachusetts Med Sch Worcester · NIH-11434593 · Data updated 2026-11-12
Researchers are studying how new mutations that occur after conception might contribute to ALS, with a focus on spinal cord motor neurons and cells from ALS patients.
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, 2026 |
| Project end | August 31, 2031 |
| Funding year | 2026 |
| Award (this year) | $542,777 |
| NIH project number | 1R01NS151040-01 |
| Project ID | NIH-11434593 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are individuals with ALS or shared datasets including ALS and non-ALS controls, plus monozygotic twins where one twin has ALS.
✕Less likely to help
Patients without ALS or related data or samples may not directly benefit from this research.
What this research studies
This project looks at whether somatic mutations—genetic changes that happen after birth—play a role in sporadic ALS. It combines clinical ALS expertise with advanced single-cell analysis to examine spinal cord tissues from ALS patients and matched controls, as well as large sequencing datasets and twins discordant for ALS. When potential deleterious mutations are found, they will be tested in laboratory models using patient-derived motor-neuron-like cells edited with CRISPR. The goal is to uncover new genetic drivers that could explain why ALS develops in people without a family history.
Why it matters
Potential benefit: If successful, this work could reveal new genetic factors behind ALS and point toward targeted therapies or personalized strategies for patients.
How similar studies have gone: Previous work has shown that somatic mutations can contribute to certain neurological disorders, suggesting potential relevance to ALS that this study will further explore.
Where this research is happening
-
Univ of Massachusetts Med Sch Worcester
Worcester, United States Active
Researchers
- Principal investigator: Lodato, Michael Anthony — Univ of Massachusetts Med Sch Worcester
- Study coordinator: Lodato, Michael Anthony
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.