Val Altounian / Science Translational Medicine

Sickle cell disease corrected by genetic modification?

The promise of a revolutionary gene-editing technology is beginning to be realized in experiments aimed at curing sickle cell disease. Scientists reported Wednesday that they used the CRISPR-Cas9 system to correct a tiny genetic mutation that causes the blood disease, which affects millions of people around the world. So far, the feat has been demonstrated only […]