A Breakthrough Study Could Lead to Synthetic DNA Therapies for Incurable Genetic Diseases
December 11, 2017
(Quartz) – Now, researchers from University College London report they’ve found a way to reverse the effect of this genetic defect—or at least slow it down—through an injection of synthetic DNA. In a small clinical trial of 46 patients, an injection of a synthetic molecule into the spinal cord appeared to stop the mutated huntingtin gene from producing the faulty protein. If it can keep the lethal protein at bay long term, the treatment could effectively cure an otherwise fatal disease.