Victoria Gray, 34-year-old mother-of-four, has volunteered to be the first American to have their genes edited with the controversial CRISPR technology to treat her SickleCell disease (SCD).
For the first time, doctors in the U.S. have used the powerful gene-editing technique CRISPR to try to treat a patient with a genetic disorder.
Gray volunteered for a trial being conducted by the Sarah Cannon Research Center in Nashville and Vertex Pharmaceuticals. The goal is to use CRISPR to edit her DNA and flip a genetic switch that is typically only ‚on‘ in newborns to make new hemoglobin, the oxygen-carrying protein.
If this works, the fetal protein might be a lifelong fix for Gray and hundreds of thousands more sickle cell sufferers around the world.
„It’s exciting to see that we might be on the cusp of a highly effective therapy for patients with sickle cell,“ says Dr. David Altshuler, Executive Vice President at Vertex Pharmaceuticals, Boston. Vertex is conducting the study with CRISPR Therapeutics of Cambridge, Mass.
The treatment is not without risks, but bioethicist Dr Arthur Caplan of New York University views it with cautious optimism.Read more
Source: Daily Mail, UK