Using CRISPR Cas9 to cure sickle cell disease

Using CRISPR Cas9 to cure sickle cell disease


(light orchestral music) – A crucial question is whether or not this gene editing treatment is lasting. We edit the cells today. Will the edited cells be around to keep you healthy years
from now, potentially? So, to test that, we edited
human cells in the laboratory. We then injected them into mice. And then we looked at
the editing of the cells four months later and asked whether or not the edited cells hung around. When we do that we find that
about 2% of the stem cells that remain in the bone marrow
after four months are edited. Which in the case of sickle
cell disease, is, um, that level of editing is likely
to have clinical benefit, but we would like to
get a little bit better than 2% in the future. As for the excitement about
the future of this technique, I’m extremely excited because, you know, we haven’t been trying this for very long. You know, we will get better. And what we have right
now is already, you know, if we can scale it up and
make sure that it works well, is already good enough to form
the basis of a clinical trial to cure sickle cell
disease with gene editing.


5 thoughts on “Using CRISPR Cas9 to cure sickle cell disease

  1. Sickle cell can be cured with hydrogen cyanide, make sure you get that whole continent while you are at it

  2. πŸ’‰πŸ’‰πŸ’‰πŸ’‰πŸ’‰πŸ’‰πŸ’‰πŸ’‰πŸ’‰πŸ’‰πŸ’‰πŸ’‰πŸ’‰πŸ’‰πŸ’‰πŸ’‰πŸ’‰πŸ’‰πŸ’‰πŸ’‰πŸ’‰πŸ’‰πŸ’‰πŸ§ͺπŸ’§πŸ’¦πŸ‘…πŸ‘„πŸ§ πŸ§¬πŸŽπŸ₯°πŸŽπŸŽπŸŽπŸŽπŸ’‰πŸ’‰πŸ’‰πŸ’‰πŸ’‰πŸ’‰πŸ’‰πŸ’‰

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