Now, they’re working to see if the MM-401 eraser technique works with human stem cells that bear some resemblance to mouse epiblast stem cells. Currently, embryos left over from infertility treatments are the only source of human embryonic stem cells. Similar attempts by other teams to restore pluripotency to mouse cells from the epiblast stem cell state have yielded far lower amounts, or nonviable cells.
The drug MM-401 targets the labels that come from the activity of a gene called MLL1. MLL1 plays a key role in the uncontrolled explosion of white blood cells that’s the hallmark of leukemia, which is why U-M researchers originally developed MM-401 to interfere with it.
Stem cells don’t harness the power of MLL1 until they’re older. The cells couldn’t continue on their journey to becoming different types of cells.
As part of the work, Sundeep Kalantry, Ph.D., an assistant professor of human genetics at U-M, showed that female stem cells treated with MM-401 had un-silenced one of their X chromosomes.