If UCSF is known for birthing the field of fetal surgery, UC Berkeley, located a short drive across the Bay Bridge, is famous in biomedical circles for pioneering CRISPR gene editing, the most powerful DNA-manipulating tool ever invented. What [Pediatric and fetal surgeon Tippi] MacKenzie envisions — the future she is now preparing for — is the convergence of these technologies into a whole new field of medicine dedicated to curing diseases before birth: fetal genome surgery. This is surgery without scalpels or sutures, just a syringe pushing particles containing CRISPR into the vein that connects a pregnant person to the fetus. Once inside the fetus’ cells, CRISPR’s molecular scissors snip away the string of problematic DNA, stopping a catastrophic genetic disease before it really starts. If successful, it would fundamentally and forever change the practice of fetal and maternal care.
This tantalizing prospect recently led the National Institutes of Health to begin funding projects that are carving a path to clinical trials. Getting permission from the Food and Drug Administration to test a fetal therapy is an enormous challenge, even more so when it involves an emergent technology like CRISPR. Yet, if anyone can do it, scientists told STAT, it’s MacKenzie.
