Stopping cancer by targeting gene transcription and RNA splicing
Official title: Targeting Transcription and Splicing in Cancer
Funded at Cold Spring Harbor Laboratory · NIH-11294223 · Data updated 2026-11-12
Researchers are developing therapies that change how cancer cells read genes and process RNA to treat aggressive breast and pancreatic cancers.
Quick facts
| Grant type | P01 program project |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Cold Spring Harbor Laboratory NIH-funded |
| Lab location | 1 site (Cold Spring Harbor, United States) |
| Project start | February 10, 1997 |
| Project end | January 31, 2028 |
| Funding year | 2026 |
| Award (this year) | $2,880,000 |
| NIH project number | 5P01CA013106-54 |
| Project ID | NIH-11294223 on NIH RePORTER |
Who could benefit from this research
✓Could help
People with basal-like (often triple-negative) breast cancer or aggressive pancreatic cancer, especially whose tumors show abnormal transcription or splicing patterns, would be the most likely candidates for related therapies or future trials.
✕Less likely to help
Patients with cancers driven by unrelated mechanisms, early-stage tumors already curable by standard care, or non-cancer conditions would be unlikely to benefit directly from this program.
What this research studies
This program studies how cancer cells rewire gene transcription and RNA splicing to help tumors start and grow. Scientists use patient-derived organoids, advanced imaging in animal models, and molecular lab methods to find vulnerabilities in tumors. They are testing antisense oligonucleotides—small pieces of genetic material—to block otherwise untreatable cancer targets and correct faulty RNA splicing. Four shared cores provide sequencing, biochemistry, imaging, and other technologies to speed development of new treatments.
Why it matters
Potential benefit: If successful, the work could lead to precise new therapies that stop or slow aggressive breast and pancreatic tumors by targeting their gene‑reading and RNA‑processing machinery.
How similar studies have gone: Antisense oligonucleotide drugs have succeeded for some genetic diseases and early cancer research is promising, but splicing‑targeted cancer treatments remain largely experimental.
Where this research is happening
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Cold Spring Harbor Laboratory
Cold Spring Harbor, United States Active
Researchers
- Principal investigator: Vakoc, Christopher — Cold Spring Harbor Laboratory
- Study coordinator: Vakoc, Christopher
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.