Protecting the striatal neurons that fail in Huntington's disease
Official title: Identifying Factors Regulating Medium Spiny Neuron Differentiation or Maintenance as Therapeutic Targets for Huntington's Disease using Induced Pluripotent Stem Cells
Funded at Buck Institute for Research on Aging · NIH-11330611 · Data updated 2026-11-12
This project looks at whether correcting early developmental problems in the striatal brain cells affected by Huntington's disease can be reversed using patient-derived stem cells and repurposed drugs.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Buck Institute for Research on Aging NIH-funded |
| Lab location | 1 site (Novato, United States) |
| Project start | September 30, 2016 |
| Project end | April 30, 2029 |
| Funding year | 2026 |
| Award (this year) | $785,686 |
| NIH project number | 5R01NS100529-08 |
| Project ID | NIH-11330611 on NIH RePORTER |
Who could benefit from this research
✓Could help
People who carry the Huntington's disease mutation or have early-stage HD would be the most relevant candidates to provide samples or be considered for follow-up studies based on these findings.
✕Less likely to help
Individuals without the HD mutation, or those in very advanced stages of neurodegeneration, are less likely to receive direct benefit from this line of work in the near term.
What this research studies
From your point of view, researchers are growing brain cells made from people with Huntington's disease using induced pluripotent stem cells (iPSCs) to model the specific neurons (medium spiny neurons, MSNs) that die in HD. They compare how these MSNs develop and which genes are turned on or off using bulk and single-cell RNA sequencing to find abnormal transcriptional programs. The team will test whether changing key MSN developmental transcription factors (for example DLX family members and BCL11B) or giving existing drugs identified by computer-based bioinformatics can restore normal MSN development and maintenance. Findings come from lab experiments on human cell models and drug screens, with the goal of identifying targets that could later be pursued in patients.
Why it matters
Potential benefit: If successful, this work could point to treatments that restore healthy development or stability of striatal neurons and slow or prevent Huntington's disease progression.
How similar studies have gone: Previous human iPSC studies have revealed developmental and transcriptional changes in HD neurons and some drug-repurposing efforts have shown promise, but targeting MSN developmental transcription factors like BCL11B is a relatively new approach.
Where this research is happening
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Buck Institute for Research on Aging
Novato, United States Active
Researchers
- Principal investigator: Ellerby, Lisa M — Buck Institute for Research on Aging
- Study coordinator: Ellerby, Lisa M
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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.