Finding new cancer targets in blood disorders caused by splicing mutations

Identifying mis-splicing-derived antigens in spliceosomal mutant myelodysplasia and targeting via T-cell receptor therapeutics

NIH-funded research Weill Medical Coll of Cornell Univ · NIH-11015772

This study is looking at how specific changes in RNA can lead to blood disorders like myelodysplastic syndromes and acute myeloid leukemia, and it aims to find new ways to create treatments that help the immune system target these changes for patients with these conditions.

Quick facts

Grant typeFellowship grant
Study typeNIH-funded research
Funding institutionWeill Medical Coll of Cornell Univ NIH-funded
Lab location1 site (New York, United States)
Project IDNIH-11015772 on NIH RePORTER

What this research studies

This research investigates how certain mutations in RNA splicing factors contribute to myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML). The team aims to identify tumor antigens generated from mis-spliced mRNAs due to these mutations, which could be targeted by T-cell receptor (TCR) therapeutics. By analyzing patient samples and using advanced techniques like RNA sequencing and immunopeptidomics, the researchers hope to develop new immunization strategies and therapies for patients with these blood disorders.

Who could benefit from this research

Good fit: Ideal candidates for this research are patients diagnosed with myelodysplastic syndromes or acute myeloid leukemia, particularly those with spliceosomal mutations.

Not a fit: Patients without these specific blood disorders or those not exhibiting spliceosomal mutations may not benefit from this research.

Why it matters

Potential benefit: If successful, this research could lead to innovative treatments that specifically target cancer cells in patients with MDS and AML.

How similar studies have performed: Previous research has shown promise in targeting mis-spliced mRNAs for immunotherapy, indicating a potential for success in this novel approach.

Where this research is happening

New York, 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.
Last reviewed 2026-06-10 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.