Improving the effectiveness of stem cell therapy for sickle cell disease
Official title: Evaluating accessory cells to improve homing and engraftment efficiency of CD34+ cells from sickle cell disease patients
Funded at New York Blood Center · NIH-10954206 · Data updated 2026-11-12
This study is looking at new ways to improve the collection and use of stem cells for gene therapy in people with sickle cell disease, making it easier for patients to get the treatment they need, especially in areas with fewer resources.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | New York Blood Center NIH-funded |
| Lab location | 1 site (New York, United States) |
| Project start | September 1, 2024 |
| Project end | July 31, 2026 |
| Funding year | 2024 |
| Award (this year) | $128,100 |
| NIH project number | 1R21HL175096-01 |
| Project ID | NIH-10954206 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals aged 21 and older who have been diagnosed with sickle cell disease and are considering gene therapy.
✕Less likely to help
Patients with other hematological disorders or those who are not eligible for stem cell therapy may not benefit from this research.
What this research studies
This research investigates ways to enhance the collection and effectiveness of hematopoietic stem cells (HSCs) for gene therapy in patients with sickle cell disease (SCD). The approach focuses on evaluating accessory immune cells that may improve the homing and engraftment of CD34+ cells, which are crucial for successful treatment. By addressing logistical challenges and improving access to gene therapy, this research aims to make treatment more feasible, especially in regions with limited resources. The study will involve innovative techniques to optimize the use of stem cells collected from patients, potentially leading to better outcomes.
Why it matters
Potential benefit: If successful, this research could significantly improve the accessibility and effectiveness of gene therapy for patients with sickle cell disease.
How similar studies have gone: Previous research has shown promise in enhancing stem cell engraftment through similar methods, indicating potential for success in this approach.
Where this research is happening
-
New York Blood Center
New York, United States Active
Researchers
- Principal investigator: Shi, Patricia Ann — New York Blood Center
- Study coordinator: Shi, Patricia Ann
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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.