In layman's terms: She had a particularly vicious form of cancer where the very cells that are supposed to protect your health -- your T-cells -- are the enemy. So they made three edits to donor cells and wiped out her compromised cells. Then gave her a bone marrow transplant afterwards to replenish her immune system with new cells.
I'm squeamish about genetic research, but this looks really good to me. I hope she recovers fully and there are no further complications, though I imagine it's too early to guess what her prognosis might be.
So there are already trials of base editing under way in sickle-cell disease, as well as high cholesterol that runs in families and the blood disorder beta-thalassemia.
That's a big deal for people with those genetic disorders.
I'm squeamish about genetic research, but this looks really good to me. I hope she recovers fully and there are no further complications, though I imagine it's too early to guess what her prognosis might be.
So there are already trials of base editing under way in sickle-cell disease, as well as high cholesterol that runs in families and the blood disorder beta-thalassemia.
That's a big deal for people with those genetic disorders.