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Doctors Use CRISPR To Treat Patient With Genetic Disorder

Posted on July 30, 2019 by Medtech[y] Staff

NPR published an exclusive interview with a patient who received the powerful gene-editing technique CRISPR to try and treat a genetic disorder. The patient, Victoria Grey, is also the first patient to be publicly identified as being involved in a study testing the use of CRISPR for genetic disease.

"It is just amazing how far things have come," says Victoria Gray, 34, of Forest, Miss. "It is wonderful," she told NPR in an exclusive interview after undergoing the landmark treatment for sickle cell disease.

Sickle cell affects millions of people around the world, of which around 100,000 are in the U.S. The company behind the treatment, CRISPR Therapeutics, announced the treatment of an anonymous patient but NPR was able to get access to Grey.

The study entails taking cells from patients' own bone marrow that's been genetically modified with CRISPR to make them product a protein that will hopefully compensate for the defective protein that causes sickle cell disease, allowing patients' to resume normal lives.

"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 Altschuler, executive vice president, global research and chief scientific officer at Vertex Pharmaceuticals in Boston. Vertex is conducting the study with CRISPR Therapeutics of Cambridge, Mass.

CRISPR Therapeutics Corporate Presentation