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Genome surgeons target genetic disease at the source

| | December 7, 2018

[Delaney Van Riper] was born with a rare genetic disease called Charcot-Marie-Tooth, or CMT, which is slowly eroding her nerve cells’ ability to ping messages back and forth between her brain and her muscles. Doing things with her hands and feet, like walking and holding a pencil, has been growing progressively harder. But last year she became one of a handful of patients whose cells are undergoing experimental Crispr procedures.

In September, UC Berkeley biochemist and Crispr pioneer Jennifer Doudna announced she was opening up a lab across the Bay to establish the epicenter of a whole new field of medicine: genome surgery.

What she envisions is the development of procedures closer to how surgeons slice out malignant tumor tissues with scalpels today. Except tomorrow’s genome surgeons will use Crispr’s molecular scissor function to remove or replace faulty genes.

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What’s the right dose? What’s the right way to calculate risks and benefits for patients? What data do you have to collect to know it’s safe and effective? Doudna and her collaborators at the Gladstone hope to answer these big questions over the next five to ten years. Their efforts are perhaps the most concentrated push in a national effort to create the tools and rules necessary for doctors to widely adopt genome editing as part of standard care.

Read full, original post: Inside the lab training genome surgeons to fight disease

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