Paired genetic weaknesses in acute myeloid leukemia
Investigating novel synthetic lethal epigenetic interactions in Acute Myeloid Leukemia
Researchers are looking for pairs of gene-related weaknesses in acute myeloid leukemia cells to find new ways to attack the cancer.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Pennsylvania NIH-funded |
| Lab location | 1 site (Philadelphia, United States) |
| Project ID | NIH-11235180 on NIH RePORTER |
What this research studies
The team uses a gene-editing method (CRISPR-Cas12a) to turn off two genes at once in leukemia cells and see which combinations stop cancer growth. Their early screens found promising pairs such as BRD9 with JMJD6 and KAT6A with JMJD6 that appear to act together to impair AML cells. They will confirm these hits in additional lab models and study how blocking both partners affects leukemia biology. The eventual aim is to prioritize paired targets that could be developed into new combination therapies for AML.
Who could benefit from this research
Good fit: Patients with acute myeloid leukemia—particularly those whose disease shows abnormal epigenetic regulation—would be the eventual candidates for follow-up therapies or trials based on this work.
Not a fit: People with other blood cancers or AML driven by non-epigenetic mechanisms are less likely to benefit from these specific epigenetic target findings.
Why it matters
Potential benefit: If successful, this work could point to new drug combinations that more effectively kill AML cells and lead to better treatments.
How similar studies have performed: Single-agent epigenetic drugs have shown limited benefit in AML, but early laboratory work and this project's preliminary data suggest that targeting paired epigenetic factors is a promising and novel approach.
Where this research is happening
Philadelphia, United States
- University of Pennsylvania — Philadelphia, United States (Active)
Researchers
- Principal investigator: Shi, Junwei — University of Pennsylvania
- Study coordinator: Shi, Junwei
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.