>Actually - more realistic would be, What if you had the disease in question and knew the cure might (then again might not) have some unknown side effect, up to

>Actually - more realistic would be, What if you had the disease in question and knew the cure might (then again might not) have some unknown side effect, up to or even exceeding a 10% chance of death. Would you take it? Yea. That exists commonly. It's called high risk surgery. This genetic disease is more like you die in ~5 years and also all the nerves start to fail including ones involving thinking, memory, personality (cerebral) Same with cancer truly. Many patients treated for childhood solid tumors go onto get blood cancers due to the treatment/chemo being genotoxic. Oh, and infertile also. So I think gene therapy should be considered totally valid when these other potentially deadly treatments (surgery, chemo) are used - all of it in deadly situations. Gene therapy also has ways to go, lentiviruses are well known to randomly integrate sometimes in the wrong spot (a tumor suppressor gene) whereas CRISPR can be more targeted.