Gene therapy offers hope for inherited blood disease
This article was originally published in Scrip
Executive Summary
Gene therapy has improved the symptoms of a patient with the genetic blood disease β-thalassaemia , giving hope that this could become a new, more convenient option to treat the genetic disorder. As reported in Nature, a patient who received treatment around 33 months ago has not required a blood transfusion – which used to be a monthly affair – for the past 21 months.