1:56 PM
Nov 17, 2016
 |  BC Innovations  |  Translation in Brief

Editing hemoglobin

Using CRISPR for sickle cell disease

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.

Matthew Porteus, an associate professor of pediatrics at Stanford University who led one of the studies, told BioCentury, “On paper or on a chalkboard it’s conceptually quite easy to think about - we should be able to purify those stem cells and correct that one variant, change it back to the version that doesn’t cause disease, and then give those cells back to cure the disease.”

Previous studies using other methods of homologous recombination, involving transcription activator-like effector nucleases (TALENs) or zinc finger nucleases (ZFNs) to modify the β-globin gene, failed to correct the gene in enough cells to treat...

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