MBNL1 identified as a potential cancer drug target for a type of childhood leukemia
Scientists from St. Jude Children's Research Hospital and collaborators have discovered that the RNA-binding protein MBNL1 promotes the growth of a form of childhood leukemia characterized by KMT2A gene rearrangements. The finding identifies MBNL1 as a promising therapeutic target for future drug development. The results were published August 7 in Science Advances.
St. Jude Children's Research Hospital scientists have identified the RNA-binding protein MBNL1 as a potential therapeutic target for a type of childhood leukemia characterized by KMT2A gene rearrangements. MBNL1 promotes the growth of this form of leukemia, making it an attractive target for drug development. Researchers discovered that MBNL1 helps maintain the expression of FLT3, a key oncogene that drives KMT2A-rearranged leukemia, by binding to a specific DNA region that regulates FLT3 expression.
In KMT2A-rearranged leukemia cells, MBNL1 binds to single-stranded DNA rather than the usual double-stranded DNA, preventing it from closing and activating FLT3 expression. Disrupting MBNL1's ability to bind DNA or its binding site reduced leukemia cell growth, suggesting that targeting MBNL1 and its DNA-binding mechanism could be a selective therapeutic strategy for this type of childhood leukemia.
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