Targeting leukemia cells with specific RNA mutations

Synthetic introns for selective targeting of RNA splicing factor-mutant leukemia

['FUNDING_R01'] · FRED HUTCHINSON CANCER CENTER · NIH-10891693

This study is exploring new treatments for acute myeloid leukemia (AML) by using special tools that can target and kill cancer cells with certain mutations, while leaving healthy cells unharmed, and so far, early results look promising!

Quick facts

Phase['FUNDING_R01']
Study typeNih_funding
SexAll
SponsorFRED HUTCHINSON CANCER CENTER (nih funded)
Locations1 site (SEATTLE, UNITED STATES)
Trial IDNIH-10891693 on ClinicalTrials.gov

What this research studies

This research focuses on developing new therapies for acute myeloid leukemia (AML) by targeting specific mutations in RNA splicing factors that are common in this disease. The approach involves creating synthetic introns that can selectively kill cancer cells with these mutations while sparing healthy cells. The research team combines expertise in cancer biology, RNA splicing, and drug delivery to design and test these innovative treatments. Preliminary results have shown promise in suppressing cancer growth in laboratory settings.

Who could benefit from this research

Good fit: Ideal candidates for this research are patients diagnosed with acute myeloid leukemia who have specific spliceosomal mutations.

Not a fit: Patients without spliceosomal mutations or those with other types of leukemia may not benefit from this research.

Why it matters

Potential benefit: If successful, this research could lead to more effective and targeted treatments for patients with AML and related blood disorders.

How similar studies have performed: Other research has shown success with similar targeted approaches in cancer treatment, indicating potential for this novel method.

Where this research is happening

SEATTLE, UNITED STATES

Researchers

About this research

  1. This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
  2. Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
  3. For full project details, budget, and progress reports, visit the official NIH RePORTER page below.

View on NIH RePORTER →

Last reviewed 2026-05-15 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.