MDM2 RNA splice variants and their role in cancer
Mdm2 Alternative Splicing in DNA Damage and Cancer
['FUNDING_R01'] · OHIO STATE UNIVERSITY · NIH-11394966
This work aims to change how the MDM2 gene’s RNA is processed to restore p53 tumor-suppressor activity and open new treatment options for cancers that express the MDM2-ALT1 form.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | OHIO STATE UNIVERSITY (nih funded) |
| Locations | 1 site (Columbus, UNITED STATES) |
| Trial ID | NIH-11394966 on ClinicalTrials.gov |
What this research studies
If you have cancer, this project studies a specific abnormal form of the MDM2 gene product (called MDM2-ALT1) that can affect the p53 tumor suppressor. Researchers will identify the proteins and RNA factors that cause cells to make this alternate MDM2 splice form during tumor development. They will use lab models and human tumor samples to test splice-switching oligonucleotides designed to shift splicing away from the harmful isoform. The goal is to see whether changing splicing can reduce the cancer-promoting effects of MDM2-ALT1.
Who could benefit from this research
Good fit: Ideal candidates would be people with tumors known to express MDM2-ALT1 (for example certain pediatric high-grade gliomas, rhabdomyosarcomas, liposarcomas, some lymphomas, or select breast cancers) or patients willing to provide tumor tissue for testing.
Not a fit: Patients whose cancers do not express MDM2-ALT1 or who have damaging mutations in the p53 gene are less likely to benefit from this approach.
Why it matters
Potential benefit: If successful, this approach could restore p53 activity and lead to a new targeted treatment for cancers driven by MDM2-ALT1.
How similar studies have performed: Splice-switching oligonucleotides have produced clinical benefits in non-cancer diseases (e.g., spinal muscular atrophy) and shown promise in preclinical cancer models, but directly targeting MDM2-ALT1 in patients is largely untested.
Where this research is happening
Columbus, UNITED STATES
- OHIO STATE UNIVERSITY — Columbus, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: CHANDLER, DAWN S — OHIO STATE UNIVERSITY
- Study coordinator: CHANDLER, DAWN S
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.
Conditions: Anti-Cancer Agents