ARTICLE | Translation in Brief
Editing hemoglobin
Using CRISPR for sickle cell disease
November 17, 2016 9:56 PM UTC
Three independent teams have demonstrated three different ways of using CRISPR to treat sickle cell disease, paving the way for correcting genetic mutations in a disease where the only long-term therapeutic option is blood transplants that have toxic side effects.
Because sickle cell disease is caused by a single mutation - a glutamate-to-valine substitution in both copies of the β-globin (HBB) gene - researchers have long considered it low-hanging fruit for correction via gene editing...