A landmark study conducted by Intellia has successfully used CRISPR-Cas9 to cut a gene in vivo out of the liver cells of people with ATTR amyloidosis. "It's a critical first step in being able to inactivate, repair or replace any gene that causes disease, anywhere in the body. This opens up the possibility to treat a wide range of diseases that we haven't been able to address so far," said Jennifer Doudna, co-founder of Intellia.
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