Altering DNA to Cure Disease

JENNIFER DOUDNA — CO-FOUNDER, INTELLIA THERAPEUTICS

In defending themselves against attacking viruses, bacteria use a naturally-occurring technique of slicing up a virus’ genetic material and pasting it into their own. Jennifer Doudna, a professor at the University of California, Berkeley and co-founder of the biotechnology firm Intellia, found she could mimic this technique to edit the DNA of humans— adding, deleting or replacing new genetic instructions for human cells. This method of gene-editing, known as CRISPR, has proven to be faster, cheaper and more accurate than other approaches. It has already shaken up the world of life sciences, leading to new treatments for genetic diseases such as sickle cell anemia and some eye and liver diseases.

For this work, Doudna and fellow researcher Emmanuelle Charpentier received the Nobel Prize in chemistry last year. This summer, CRISPR was used to treat a rare disease directly through an intravenous infusion, rather than having to remove cells, manipulate them and reintroduce them to the body, as current methods required. “It’s a clear indication that a new era of genetic medicine is now upon us,” says Doudna. “I’m hopeful that over the next few years, these results can be replicated to other target disease areas and organs such as the brain and heart, where molecular delivery is more difficult.”