In DepthBiomedicine

Tweaking genes with CRISPR or viruses fixes blood disorders

See allHide authors and affiliations

Science  11 Dec 2020:
Vol. 370, Issue 6522, pp. 1254-1255
DOI: 10.1126/science.370.6522.1254

You are currently viewing the summary.

View Full Text

Log in to view the full text

Log in through your institution

Log in through your institution

Summary

It is a double milestone: new evidence that cures are possible for many people born with sickle cell disease and another serious blood disorder, beta-thalassemia, and a first for the genome editor CRISPR. Two strategies for directly fixing malfunctioning blood cells have dramatically improved the health of a handful of people with these genetic diseases, teams reported online on 4 December in The New England Journal of Medicine and 1 day later at the American Society of Hematology meeting. One relies on CRISPR, marking the first inherited disease clearly helped by the powerful tool created just 8 years ago. And both treatments are among a wave of genetic strategies poised to widely expand who can be freed of the blood disorders.

View Full Text

Stay Connected to Science