Researchers have used a highly precise genome-editing technology called base editing to make hundreds of direct edits to blood stem cells from patients’ bone marrow.
Their work, published today in Cell, is the first time that such high-throughput base editing, which can make many single-base substitutions in DNA in many cells at once, has been applied to blood stem cells. The research team showed how such single-nucleotide changes in genes can affect the biology of blood cells and contribute to the treatment of diseases including leukemia and sickle cell disease. The findings suggest how the technology can help scientists learn more about the role of gene variants in disease in multiple cell types.
“Our approach allows us to understand not just whether a particular gene might be implicated in human disease, but exactly how individual changes to that gene are playing out at a molecular level,” said Vijay Sankaran, a senior author of the new study and an associate member at the Broad Institute of MIT and Harvard. “This finer resolution gives us a new roadmap for how diseases occur and how to treat them.”
