Although the tests are for the prevention of a life-threatening metabolic disorder,
the prenatal animal testing may be offering the potential to treat many human congenital diseases before birth.
The research, which comes from Children's Hospital of Philadelphia (CHOP) and the Perelman School of Medicine at the University of Pennsylvania, is published in Nature Medicine. It offers proof-of-concept for prenatal use of a sophisticated, low-toxicity tool that efficiently edits DNA building blocks in disease-causing genes.
Using both CRISPR-Cas9 and base editor 3 . . .