Scientific Advances: Base editing in hematopoietic stem cells rescues sickle cell disease

In the lab of David Liu, a CRISPR base editor was successfully applied to correct mutations in the β-globin gene HBB.

Newby, G.A., Yen, J.S., Woodard, K.J. et al. (2021) Base editing of haematopoietic stem cells rescues sickle cell disease in mice. Nature. https://doi.org/10.1038/s41586-021-03609-w

Keywords: CRISPR, Base Editors, sickle cell disease

Questions? Email: crispr@amsterdamumc.nl