Machine-designed polymers to deliver gene-editing tools into blood stem cells
Official title: Machine-guided design of chaperone-mimetic polymeric carriers for ribonucleoprotein delivery
Funded at Colorado School of Mines · NIH-11321645 · Data updated 2026-11-12
This project uses AI to design polymer carriers that help deliver gene-editing proteins into blood stem cells more safely and affordably.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Colorado School of Mines NIH-funded |
| Lab location | 1 site (Golden, United States) |
| Project start | April 15, 2025 |
| Project end | March 31, 2028 |
| Funding year | 2026 |
| Award (this year) | $182,581 |
| NIH project number | 5R21EB036183-02 |
| Project ID | NIH-11321645 on NIH RePORTER |
Who could benefit from this research
✓Could help
People with inherited blood disorders or those who might be candidates for hematopoietic stem cell gene therapies (for example, sickle cell disease or thalassemia) are most likely to benefit from advances made here.
✕Less likely to help
Patients with conditions unrelated to blood or stem-cell gene therapies, or those seeking immediate clinical treatment, are unlikely to benefit directly from this early-stage lab research.
What this research studies
Researchers will use machine learning to design polymers that mimic cellular chaperones and protect ribonucleoprotein (RNP) gene-editing payloads from misfolding inside cells. These chaperone-mimetic polymers are tested in lab models of hematopoietic stem and progenitor cells (HSPCs) to improve delivery efficiency and reduce harmful off-target edits. The team will apply transfer learning to adapt design models for rare or hard-to-grow blood stem cells, aiming to lower reliance on costly RNP production and on harsh delivery methods like electroporation. Work is preclinical and performed in laboratory settings focusing on cellular and molecular outcomes.
Why it matters
Potential benefit: If successful, this could make gene-editing treatments for blood disorders safer, more precise, and less expensive.
How similar studies have gone: Electroporation and nanoparticle approaches have enabled gene editing in blood cells, but the combination of machine-guided design and chaperone-mimetic polymers is a new and largely untested strategy.
Where this research is happening
-
Colorado School of Mines
Golden, United States Active
Researchers
- Principal investigator: Kumar, Ramya — Colorado School of Mines
- Study coordinator: Kumar, Ramya
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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.