While a cure has so far eluded researchers, advances in human gene-editing technology are poised to put SCD in check. Vertex Pharmaceuticals and the CRISPR Therapeutics companies have developed a therapy that allows doctors to edit stem cells to produce fetal instead of adult mutant hemoglobin. The engineered stem cells are returned to the patient, who then manufactures fetal hemoglobin in their own blood cells, potentially curing the disease. Clinical trials to evaluate the safety and efficacy of the treatment have just begun.
On this episode of Talking Biotech, Dr. Brenda Eustace, director of discovery research at Vertex, joins University of Florida plant geneticist Kevin Folta to discuss this promising sickle cell treatment that could bring needed relief to millions of people worldwide.
The Talking Biotech podcast, produced by Kevin Folta, is available for listening or subscription: