A US start-up has successfully treated the first patients using a Crispr gene-editing therapy directed inside the body to an internal organ.
The early trial data from Intellia Therapeutics, co-founded by Nobel Prize winner Jennifer Doudna, marked a breakthrough for Crispr-based treatments, showing scientists had overcome challenges that had previously restricted the technology’s use to editing cells outside the body or in the eye.
The Boston-based start-up, working with biotech company Regeneron, treated transthyretin amyloidosis, a devastating disease in which a build-up of a problematic protein hits a patient’s heart and nervous system, cutting their life expectancy.
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