Super Hopes for Supercells

US researchers reported promising early results treating sickle cell disease using CRISPR technology this week.
 
Edited “super cells” in a patient appear to be responding as hoped—mass producing a critical protein lacking in most patients with the debilitating disease. Still, long-term success is unknown.

Vertex Pharmaceuticals and CRISPR Therapeutics—the study’s 2 sponsor companies—also saw early success treating a German patient with beta thalassemia, a similar disorder. 

“This is the first evidence that the new CRISPR technology has the potential to be curative for serious genetic diseases,” says Vertex’s David Altshuler. 

NPR

Comments +

0 comments

Post a Comment

Restricted HTML

  • Lines and paragraphs break automatically.
  • Web page addresses and email addresses turn into links automatically.
CAPTCHA
This question is for testing whether or not you are a human visitor and to prevent automated spam submissions.
Back to top