Using viral particles to modify blood stem cells for treating blood disorders
Official title: In vivo hematopoietic stem cell genetic modification by viral particles
Funded at Boston Children's Hospital · NIH-11189727 · Data updated 2026-11-12
This study is exploring a new way to use viral particles to change blood stem cells inside the body to help treat serious blood disorders like X-linked Severe Combined Immunodeficiency and Sickle Cell Disease, making the treatment safer and easier for more patients.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Boston Children's Hospital NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project start | September 9, 2024 |
| Project end | May 31, 2028 |
| Funding year | 2025 |
| Award (this year) | $874,907 |
| NIH project number | 5R01HL172489-02 |
| Project ID | NIH-11189727 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research would be individuals diagnosed with severe monogenic blood disorders such as X-SCID or Sickle Cell Disease.
✕Less likely to help
Patients with blood disorders that are not monogenic or those who do not have a suitable genetic target for this therapy may not benefit from this research.
What this research studies
This research focuses on developing a new method to modify hematopoietic stem cells (HSCs) using viral particles to treat severe blood disorders like X-linked Severe Combined Immunodeficiency and Sickle Cell Disease. The approach aims to simplify the gene therapy process by allowing for in vivo modifications, which means that the treatment can be done inside the body rather than requiring complex procedures outside the body. This could make the therapies safer, more effective, and easier to produce for a larger number of patients.
Why it matters
Potential benefit: If successful, this research could provide a more accessible and safer gene therapy option for patients suffering from severe blood disorders.
How similar studies have gone: Other research has shown promising results with similar gene therapy approaches, indicating a strong potential for success with this novel method.
Where this research is happening
-
Boston Children's Hospital
Boston, United States Active
Researchers
- Principal investigator: Bauer, Daniel Evan — Boston Children's Hospital
- Study coordinator: Bauer, Daniel Evan
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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.