Protecting blood stem cells from harmful aldehydes in Fanconi anemia
Protection of hematopoietic stem progenitor cells from endogenous DNA damage
Looks for ways to boost blood stem cells' ability to clear harmful aldehydes to help people with Fanconi anemia avoid bone marrow failure and cancer.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Johns Hopkins University NIH-funded |
| Lab location | 1 site (Baltimore, United States) |
| Project ID | NIH-11323509 on NIH RePORTER |
What this research studies
Researchers will study human blood-forming stem cells to understand how enzymes called ALDH and ADH protect DNA from damage by reactive aldehydes. They will use single-cell CRISPR activation and CRISPR knockout screens in human cells transplanted into mice to find genes that increase aldehyde detoxification. The team will test activating those protective genes and delivering functional enzymes via extracellular vesicles to stop aldehyde-induced DNA damage. The goal is to identify strategies that could be developed into treatments to prevent bone marrow failure and blood cancers in people with Fanconi anemia.
Who could benefit from this research
Good fit: Ideal candidates are people diagnosed with Fanconi anemia or those at high risk of bone marrow failure who may be interested in donating samples or participating in future trials.
Not a fit: People without Fanconi anemia or those seeking an immediate clinical treatment are unlikely to receive direct benefit from this laboratory-focused research at this time.
Why it matters
Potential benefit: If successful, this work could lead to new therapies that lower harmful aldehyde levels and prevent bone marrow failure and blood cancers in people with Fanconi anemia.
How similar studies have performed: Enzyme-targeting and gene-activation approaches are relatively novel for Fanconi anemia, with limited prior clinical success but encouraging preclinical evidence in related DNA-repair fields.
Where this research is happening
Baltimore, United States
- Johns Hopkins University — Baltimore, United States (Active)
Researchers
- Principal investigator: Jung, Moonjung — Johns Hopkins University
- Study coordinator: Jung, Moonjung
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.