Thalassaemia Gene Therapy Trial Shows ‘Encouraging’ Results

April 24, 2018

(BBC) – Scientists say they are “excited” by the results of a gene therapy trial for the inherited blood disorder beta-thalassaemia, which reduced the need for blood transfusions. In 22 patients, blood stem cells were extracted, treated and re-introduced to stimulate red blood cell production. Fifteen patients were able to stop transfusions altogether while others needed fewer of them.