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.
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.