Raza headshotInvestigator: Dr. Azra Raza and Dr. Naomi Galili

Research Institution: Columbia University

Disease: Myelodysplastic Syndrome

Research Description: Myelodysplastic syndrome (MDS) is a group of bone marrow disorders where the bone marrow cannot produce enough healthy cells. This leads to low blood cell counts and a higher risk for developing acute myeloid leukemia. In the last couple of years, our understanding of the molecular genetic basis of MDS has increased exponentially. This research will look at a specific type called of MDS called RARS, which is found in 10-15% of all MDS patients. The research team will identify mutations unique to MDS‐RARS patients, and use new technologies, such as CRISPR CAs9 gene editing, to see if altering certain genes can increase cell survival. They expect that these results will provide insights into repurposed therapies that can be rapidly developed and used in the clinic.

CWR fuding role: Primary funder 

Start date: August 1, 2012


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