Targeting genetic defects in a type of blood disorder called myelodysplastic syndrome.

Who could benefit from this research

What this research studies

This research investigates how mutations in the SF3B1 gene contribute to myelodysplastic syndromes (MDS), a condition that leads to bone marrow failure. The study focuses on understanding the role of these mutations in disrupting RNA transcription and how this affects blood cell formation. By exploring the molecular mechanisms involved, the research aims to identify potential therapeutic targets that could improve treatment options for patients with MDS. Patients may benefit from new therapies developed based on the findings of this research.

Why it matters

Where this research is happening

• Yale University
  New Haven, United States Active

Researchers

• Principal investigator: Boddu, Prajwal — Yale University
• Study coordinator: Boddu, Prajwal

Related research

Other NIH-funded projects on the same topics.

• Therapeutic targeting of transcription-epigenetic defects in SF3B1-mutant MDS

  A training-focused grant to help a clinician-scientist develop therapies for adults with a common bone marrow failure condition called SF3B1-mutant myelodysplastic syndrome (MDS).

  NIH research Yale University · New Haven, United States · NIH-11332844

• Understanding how SF3B1 mutations affect blood cell development in myelodysplastic syndromes.

  This study is looking at how changes in a specific gene (SF3B1) affect blood cell production in people with myelodysplastic syndromes (MDS), using advanced techniques to understand these changes at the single-cell level, which could help improve treatments for the condition.

  NIH research Weill Medical Coll of Cornell Univ · New York, United States · NIH-11014391

• Interferon-γ plus donor immune cells to fight AML/MDS after partial donor transplant

  This project tests a therapy that adds a signaling protein, IFN-γ, with donor immune cells to help treat relapsed AML/MDS after a haploidentical stem cell transplant.

  NIH research University of Pittsburgh at Pittsburgh · Pittsburgh, United States · NIH-11453101

• Whole-genome sequencing to refine risk in myelodysplastic syndromes (MDS) for older adults

  This project uses clinical whole-genome sequencing to improve genetic profiling and better predict how MDS will progress in older patients.

  NIH research Washington University · Saint Louis, United States · NIH-11415218

• Targeting gasdermin D to treat myelodysplastic syndromes in people with bone marrow inflammation

  A research project aims to find new ways to treat myelodysplastic syndromes by blocking a protein (gasdermin D) involved in harmful bone marrow inflammation.

  NIH research Northwestern University · Chicago, United States · NIH-11371104

• Understanding why some MDS patients relapse after a stem cell transplant

  A study of patients with myelodysplastic syndromes after stem cell transplant to learn the cellular changes and gene activity that lead to relapse, with the goal of guiding future treatments.

  NIH research Medical College of Wisconsin · Milwaukee, United States · NIH-11350441

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.