4 ms·

Is it enough to edit the genes or is there a need to activate some kind of regenerative process? The body is regenerating all the time but larger changes might

• adventured 9y ago
  The very earliest targets are ideally large gene punch-out targets, using Cas9. For example that's what Editas is targeting with their first retina program. To deal with complex, higher order genetic diseases (which is also where the money will be in the field) you'll have to be able to do inserting of healthy replacement genes. Cas9 is not very good at that as of now, it's like using a mallet to tie a fishing line. There is an immense amount of effort going into trying to shoehorn Cas9 into being better at that. Other options such as Cpf1 (and possibly one day CasX/CasY from Berkeley) have been shown to be far superior at more advanced editing.