A Crack In Creation: A Nobel Prize Winner's Insight into the Future of Genetic Engineering

by Jennifer A. Doudna

For example, in one study from Harvard Medical School, Benjamin Ebert’s team of researchers wanted to understand the genetic causes of acute myeloid leukemia, a cancer of white blood cells. By programming CRISPR to edit different genes—using a different guide RNA to match each one—they set out to knock out eight candidate genes. This kind of multiplexed gene editing would have been unthinkable before, but with CRISPR, it was straightforward. After editing the genes in blood stem cells in all different combinations and then injecting the cells back into the bloodstream of live mice, the researchers waited to see which animals came down with acute leukemia (a sort of reverse ex vivo application). Then, by cross-checking those animals with the genes that had been inactivated with CRISPR, Ebert’s team deduced the exact constellation of gene mutations necessary and sufficient to promote leukemia. Experiments like these are proving invaluable for advancing human cancer research.