Rare genetic diseases are challenging for patients and their families\u2014made all the more overwhelming because symptoms tend to appear soon after birth.<\/p>\n
To date, there haven\u2019t been many reliable treatment options for these babies. The few that do exist involve invasive and risky procedures that don\u2019t often have a high rate of success.<\/p>\n
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\u2014plans for which were announced July 8\u2014is a collaboration between Jennifer Doudna, director of the Innovative Genomics Institute at the University of California, Berkeley who also earned the Nobel Prize for her work in co-discovering the gene-editing technique CRISPR, and Dr. Priscilla Chan, co-CEO and co-founder of the Chan Zuckerberg Initiative.<\/p>\n
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Supported by $20 million from the Chan Zuckerberg Initiative, the center focuses on treating rare genetic diseases in children, starting with a group of eight kids who will enroll in a clinical trial to access a CRISPR therapy designed specifically for them.<\/p>\n