The acronym is CRISPR: it stands for “clustered regularly interspaced short palindromic repeats.” It refers to a phenomenon and related technique by which scientists have already created genetically engineered laboratory animals for research purposes, and could one day be able to “edit” the human genome by replacing disease-causing genetic sequences with benign counterparts. According to a provocative New York Times article published this week, scientists in the Netherlands have shown in the laboratory that they can replace a mutation that causes cystic fibrosis. Theoretically, therapies based on CRISPR research could lead to embryonic genetic alterations that prevent CF and other genetic diseases. The popular press has already coined the term “designer babies” for the phenomenon.
As with stem cell therapy, the therapeutic possibilities are exciting—and the ethical issues are sure to be hotly debated. Scientists’ ability to make precise genetic changes in humans will doubtless have legislative and regulatory implications down the road as well.
“It does make it easier to genetically engineer the human germ line,” says Craig C. Mello, PhD, of the University of Massachusetts Medical School. The term germ line refers to the fact that such genetic engineering could lead to alterations that could be passed on to future generations. And this kind of therapy may be quickly moving beyond the realm of theory to that of application. At least three start-ups have recently been formed to provide this service: Cambridge, MA-based Editas Medicine, Caribou Biosciences in Berkeley, CA, and Crispr Therapeutics in London.
