“A new center in San Francisco will offer tailor-made CRISPR therapies to cure children with rare diseases. The Center for Pediatric CRISPR Cures, announced in June, brings together pediatrician Priscilla Chan, co-founder of the Chan Zuckerberg Initiative (CZI), and Nobel laureate Jennifer Doudna of the University of California, Berkeley. The new center builds on the successful treatment whereby baby KJ was cured of a rare and life-threatening metabolic disorder caused by carbamoyl phosphate synthetase 1 deficiency using a personalized CRISPR therapy. With $20 million in support from the CZI, the new center aims to extend CRISPR’s therapeutic impact to thousands of rare Mendelian disorders.”

From Nature.