Scientists engineered stem cells to better understand the mechanisms behind a form of leukemia caused by changes in a key gene, according to a study led by Mount Sinai researchers and published online today in the journal Cell Reports.
Past work had established that inherited changes in the DNA code for the gene PTPN11 cause Noonan syndrome, a genetic disease that comes with a high risk for the blood cancer called juvenile myelomonocytic leukemia (JMML). The mechanisms behind the disease, and what influences its severity, were unknown going into the current study.