Jun 26, 2021

CRISPR injected into the blood treats a genetic disease for first time

Posted by in categories: biotech/medical, genetics

Now, in a medical first, researchers have injected a CRISPR drug into the blood of people born with a disease that causes fatal nerve and heart disease and shown that in three of them it nearly shut off production of toxic protein by their livers.

Novel treatment using messenger RNA sharply cuts production of mutant liver protein, although it’s too early to show patients with rare condition benefit.

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