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Scientists find potential treatment for pre-malignant bone marrow disorders

Research finds key driver of myelodysplasia syndromes in blood stem cells.

By Amy Wallace
Researchers at Cincinnati Children's Hospital have developed potential treatment targets for pre-malignant bone marrow disorders. DarkoStojanovic/PixaBay
Researchers at Cincinnati Children's Hospital have developed potential treatment targets for pre-malignant bone marrow disorders. DarkoStojanovic/PixaBay

CINCINNATI, Dec. 29 (UPI) -- Researchers at Cincinnati Children's Hospital Medical Center have found a new way to target the treatment of pre-malignant disorders called myelodysplasia syndromes, or MDS.

MDS is a group of pre-malignant disorders in bone marrow that inhibit the production of healthy blood cells and can lead to acute myeloid leukemia, or AML.

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Researchers found that over-expression of a protein called TRAF6 in blood cells drives the onset of MDS.

"We found that TRAF6 over-expression in mouse hematopoietic stem cells results in impaired blood cell formation and bone marrow failure," Daniel Starczynowski, Ph.D., cancer biologist in the Division of Experimental Hematology and Cancer Biology at Cincinnati Children's Hospital and lead author of the study, said in a press release. "Based on our paper, a number of therapeutic approaches can be tested and directed against TRAF6 and other related proteins responsible for MDS."

The team used laboratory mouse models and human MDS/AML samples to identify a new substrate of TRAF6 called hnRNPA1, an RNA binding protein. They discovered molecular interactions with Cdc42, a protein that helps regulate cells found in cancer.

The findings could lead to potential treatment targets for cases of MDS caused by over-expression of TRAF6.

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Researchers also were able to identify the new molecular targets by conducting a global proteomic analysis of human leukemia cells. This revealed a new and critical immune-related function for TRAF6.

The study was published in Nature Immunology.

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