Genetic engineering support for blood and heart disease
CORE 2: Genetic Engineering Core
The team builds and improves genetic tools to change blood-forming stem cells and create better models to learn how blood and bone marrow problems relate to heart disease.
Quick facts
| Grant type | P01 program project |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Massachusetts General Hospital NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project ID | NIH-11269197 on NIH RePORTER |
What this research studies
This core develops and optimizes genome and epigenome editing methods to modify hematopoietic stem and progenitor cells (HSPCs) and to generate new mouse models. The core supports multiple project teams by designing in silico guides, running laboratory editing and validation experiments, and producing engineered cells or animals for follow-up studies. Work is primarily preclinical, using cell-based editing and animal models to probe how blood-forming cells influence cardiovascular disease. The core speeds up other projects by providing standardized, validated genetic-engineering services and expertise.
Who could benefit from this research
Good fit: People with blood or bone marrow disorders and those with cardiovascular disease are the patient groups most likely to benefit from discoveries enabled by this core, though the core itself focuses on lab work rather than enrolling patients.
Not a fit: Individuals looking for immediate treatments or people with conditions unrelated to blood or heart disease are unlikely to receive direct benefit from this core's activities.
Why it matters
Potential benefit: If successful, the work could reveal new biological pathways linking blood and bone marrow to heart disease and point to targets for future diagnostics or treatments.
How similar studies have performed: Genome and epigenome editing in blood stem cells and animal models has produced promising preclinical findings, but translating those approaches into approved human therapies remains early and ongoing.
Where this research is happening
Boston, United States
- Massachusetts General Hospital — Boston, United States (Active)
Researchers
- Principal investigator: Kleinstiver, Benjamin Peter — Massachusetts General Hospital
- Study coordinator: Kleinstiver, Benjamin Peter
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.