Engineered stem cells that deliver BDNF and gene-editing for Huntington's disease
MSCS ENGINEERED TO PRODUCE BDNF AND GENE EDITING CARGO FOR THE TREATMENT OF HUNTINGTON'S DISEASE
['FUNDING_R01'] · UNIVERSITY OF CALIFORNIA AT DAVIS · NIH-11314616
This work develops implantable human stem cells that release a protective brain growth factor (BDNF) and carry gene-editing tools to lower the harmful mutant huntingtin in adults with Huntington's disease.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UNIVERSITY OF CALIFORNIA AT DAVIS (nih funded) |
| Locations | 1 site (DAVIS, UNITED STATES) |
| Trial ID | NIH-11314616 on ClinicalTrials.gov |
What this research studies
Researchers are modifying human mesenchymal stem/stromal cells (MSCs) to secrete Brain‑Derived Neurotrophic Factor (BDNF) and to carry a novel xCas9‑based gene repression payload that lowers mutant huntingtin production. The team has shown in lab models that MSCs deliver beneficial factors and that the xCas9‑KRAB platform can reduce huntingtin expression. They are testing delivery routes (including injection into the cisterna magna as a model for CSF/spinal delivery) and optimizing the combined cell-and-gene therapy product in preclinical studies. The goal is to develop a treatment that could be moved into human trials for people with Huntington's disease.
Who could benefit from this research
Good fit: Ideal candidates for future trials would be adults with genetically confirmed Huntington's disease who meet safety and surgical eligibility criteria and are suitable for CSF or brain‑directed cell therapies.
Not a fit: People without the HD mutation, those with contraindications to neurosurgical or CSF delivery, or those with very advanced neurodegeneration may not benefit from this approach.
Why it matters
Potential benefit: If successful, this approach could protect vulnerable brain cells and reduce mutant huntingtin levels, potentially slowing disease progression and improving symptoms for people with Huntington's disease.
How similar studies have performed: BDNF delivery and huntingtin‑lowering strategies have shown benefit in animal models, but combining MSC‑delivered BDNF with xCas9 gene repression is a novel approach with limited or no human trial data yet.
Where this research is happening
DAVIS, UNITED STATES
- UNIVERSITY OF CALIFORNIA AT DAVIS — DAVIS, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: FINK, KYLE — UNIVERSITY OF CALIFORNIA AT DAVIS
- Study coordinator: FINK, KYLE
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.