Using synthetic introns to target leukemia with RNA splicing mutations
Synthetic introns for selective targeting of RNA splicing factor-mutant leukemia
Researchers are creating genetic switches called synthetic introns that turn treatments on only in leukemia cells with specific RNA splicing mutations.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Fred Hutchinson Cancer Center NIH-funded |
| Lab location | 1 site (Seattle, United States) |
| Project ID | NIH-11159644 on NIH RePORTER |
What this research studies
This team is designing 'synthetic introns'—small genetic switches that exploit abnormal RNA splicing in some leukemias to activate therapeutic genes only in cancer cells. They will test these synthetic introns in laboratory models and in animals to confirm they cause drug or toxic protein expression selectively in mutated cells while sparing healthy cells. The work combines RNA biology, functional genomics, and drug-delivery engineering to optimize both the genetic switches and how they are delivered to bone marrow and leukemia cells. If the preclinical results are strong, the approach would be advanced toward early human testing for patients with spliceosomal-mutant blood cancers.
Who could benefit from this research
Good fit: Ideal candidates would be people with acute myeloid leukemia or related myelodysplastic syndromes whose cancer cells carry RNA splicing-factor (spliceosomal) mutations.
Not a fit: Patients whose leukemia does not carry splicing-factor mutations or those with unrelated cancer types are unlikely to benefit from this approach.
Why it matters
Potential benefit: If successful, this could produce therapies that selectively kill leukemia cells with spliceosomal mutations while leaving normal cells unharmed.
How similar studies have performed: Preclinical work, including a 2022 Nature Biotechnology report, showed synthetic introns can selectively target cancer cells in animals, but clinical testing in patients has not yet been established.
Where this research is happening
Seattle, United States
- Fred Hutchinson Cancer Center — Seattle, United States (Active)
Researchers
- Principal investigator: Bradley, Robert K — Fred Hutchinson Cancer Center
- Study coordinator: Bradley, Robert K
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.