CRISPR cures for kids? A new center is born!

Jennifer Doudna and Priscilla Chan

Last May, the case of baby KJ made headlines: the child, suffering from a severe metabolic disorder, received a therapy developed specifically for him in just six months. The rapid improvement in his condition and his discharge from the hospital left the rare disease community with a pressing question: was this an unrepeatable one-off, or a replicable model of intervention? The right answer might be the latter, as demonstrated by the launch of the Center for Pediatric CRISPR Cures in California. This new center, to be led by Fyodor Urnov, begins with the mission of developing customized genome-editing treatments for eight young patients with congenital metabolic and immune system disorders.

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CRISPR-enabled carbon capture. What’s up?

Credit CSRWire

The Innovative Genomics Institute runs a program aiming to “supercharge plants and soils to remove carbon from the atmosphere” with the help of CRISPR and funding from the Chan Zuckerberg Iniziative. I asked Andy Murdock, communications director at IGI, three questions to update the picture. Please see his answers below.

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BioRxiv: an instant quality check for CRISPR science (with a little help from Zuckerberg)

bioRxiv

It was August 2 when Nature published the latest stunning study, introducing to the world the first human embryos edited in the US by Shoukhrat Mitalipov. Not even a month had passed, and on August 28, those results have been challenged on a much younger and quick medium: the bioRxiv pre-print website. I felt like a déjà vu happening. It reminded me of the Nature Methods study questioning CRISPR’s precision in June. Within three weeks bioRxiv has already challenged the controversial data about off-target mutations by posting two critical analyses which soon became three. In short, this server is rewriting a part of CRISPR’s science and it is becoming an emergency tool for correcting mistakes that, inevitably, sometimes tarnish the most respected peer-reviewed publications. How does it work? Continue reading