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.
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Intellia is looking to edit bone marrow to treat blood-based diseases without transplanting cells, including working with the Bill & Melinda Gates Foundation to treat patients in Africa with sickle cell disease.
There is a subtlety in the first quoted paragraph. It's not the first time that CRISPR has been used inside the body. Editas Medicine, co-founded by Jennifer Doudna as well, achieve so in March last year, targeting blindness caused by Leber congenital amaurosis.