Sickle cell disease corrected by genetic modification?

Val Altounian / Science Translational Medicine
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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 in human cells that were confined to laboratory dishes. But in a promising step, the researchers used the same DNA-editing technique to alter human cells that were transfused into mice. After 16 weeks, the mice still had cells that contained the edited gene, according to a study in the journal Science Translational Medicine.

“What we have right now, if we can scale it up and make sure it works well, is already enough to form the basis of a clinical trial to cure sickle cell disease with gene editing,” said study leader Mark DeWitt, a postdoctoral fellow at UC Berkeley’s Innovative Genomics Initiative.


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