It’s not using CRISPR – not yet approved for use within a human body – but zinc-finger nucleases (ZFN) to do gene editing. In this case, on a gentleman with Hunter Disease. Why mention it? I like the description of the actual treatment, one of the few attempts at using gene editing on a living human body, as found on D-brief, by Nathaniel Scharping:
Through an IV, billions of copies of a harmless virus loaded with instructions for two ZFN proteins and a gene to produce the correct enzyme were delivered to Madeux’s bloodstream. The virus was programmed to insert itself just into liver cells to ensure that it didn’t begin to edit anywhere else. Once a cell has been implanted with the virus, it begins to make copies of the ZFN proteins and the correct gene. The two proteins then cut apart the DNA at a specific point and insert the therapeutic gene, after which the cell’s natural repair mechanisms bind the double helix back together and the gene can get to work.
