The $20 Million Bet on CRISPR to Cure Rare Childhood Diseases

Rare genetic diseases are challenging for patients and their families—made all the more overwhelming because symptoms tend to appear soon after birth. To date, there haven’t been many reliable treatment options for these babies. The few that do exist involve invasive and risky procedures that don’t often have a high rate of success. But there is a new source of hope for many of these families: the Center for Pediatric CRISPR Cures at the University of California San Francisco. The center—plans for which were announced July 8—is a collaboration between...