CLINICAL TRIAL SHOWS GENE EDITING WORKS FOR Β-THALASSAEMIA, TOO

Summary from the TLDR newsletter

β-Thalassaemia, a disease closely related to sickle-cell anemia, is caused by a large range of mutations, so it can't be fixed with a single editing system. Researchers took an approach that involved reactivating the fetal version of the gene, which has a higher affinity for oxygen than normal hemoglobin. Editing the gene in patient stem cells and transplanting them back resulted in the patients' hemoglobin levels rising. All patients in the trial saw side effects, but they met the trial's key success metric of going over six months without needing a transfusion to control their disease.