[firejake308]

It's reasonable to expect small improvements in the risk profile, but I think blood cancer is going to be a side effect for any drug following this basic idea. You will always need some chemo to destroy the defective blood-making stem cells before replacing them with the genetically-modified blood-making stem cells, and any chemo that is strong enough to kill all of the blood-making stem cells in your body is necessarily going to have a risk of damaging healthy cells and turning them into pre-cancer cells. So the risk can be reduced but probably not eliminated.

[eszed]

That makes sense. Thank you.

In theory, could a separate gene therapy target and knock out the stem cells that carry the mutation?