In a landmark publication detailing the initial results of an early phase clinical trial, scientists describe the use of base editing – a new form of gene therapy – to successfully treat patients with sickle cell disease. In contrast to conventional gene editing strategies which create a cut or break in the double strand of DNA, base editing uses enzymes to correct a single misspelled base of a gene. Read more about the exciting results from the initial group of treated patients: https://hubs.ly/Q030fYdq0 #GeneTherapy #SickleCellDisease #Genomics #PrecisionMedicine
