CRISPR takes on the challenge of rare genetic 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 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.

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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.

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