Isolating stem cells from sickle cell disease patients for gene therapy
Official title: Precision Apheresis: stem cell isolation from patients with sickle cell disease for gene therapy using high-throughput microfluidics
Funded at Massachusetts General Hospital · NIH-10899569 · Data updated 2026-11-12
This study is working on a new way to collect important stem cells from the blood of people with sickle cell disease, which could help improve gene therapy treatments by making sure we get more of these valuable cells.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Massachusetts General Hospital NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project start | August 5, 2023 |
| Project end | July 31, 2028 |
| Funding year | 2024 |
| Award (this year) | $137,183 |
| NIH project number | 5K25HL169816-02 |
| Project ID | NIH-10899569 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with sickle cell disease who require stem cell therapy.
✕Less likely to help
Patients who do not have sickle cell disease or those who are not candidates for stem cell therapy may not benefit from this research.
What this research studies
This research focuses on developing a new technology to efficiently isolate hematopoietic stem and progenitor cells (HSPCs) from the blood of patients with sickle cell disease. By using a microfluidic device, the study aims to recover a high percentage of valuable stem cells from apheresis products, which are crucial for successful gene therapy. The approach involves genetic editing of these isolated stem cells and testing their effectiveness in animal models. This innovative method seeks to overcome current challenges in stem cell purification that can lead to significant cell loss.
Why it matters
Potential benefit: If successful, this research could significantly improve the availability and quality of stem cells for gene therapy in sickle cell disease patients, potentially leading to better treatment outcomes.
How similar studies have gone: Other research has shown promise in using microfluidic technologies for cell isolation, indicating potential success for this novel approach.
Where this research is happening
-
Massachusetts General Hospital
Boston, United States Active
Researchers
- Principal investigator: Mishra, Avanish — Massachusetts General Hospital
- Study coordinator: Mishra, Avanish
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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.