Emanuele and Erika Guarini are brother and sister. They were treated for thalassemia respectively in November 2020 and August 2021 by the team of Franco Locatelli at Bambin Gesù hospital in Rome. Before the CRISPR-based treatment, they needed a blood transfusion every 15-20 days (source La Repubblica).
Tag Archives: thalassemia
Three hopes for CRISPR
Off-target paper retraction: Nature Methods has retracted a controversial study questioning CRISPR precision, after its authors admitted they were probably wrong. This blog’s wish is that future studies on CRISPR flaws and virtues are as reliable as the genome-editing technique.
Deregulation statement: US Secretary of Agriculture Sonny Perdue has announced that USDA will not regulate edited plants as long as they could have been created through conventional breeding. Let’s hope Europe will follow the example.
Gene therapy going “organic”: that’s the hope expressed by Merlin Crossley, when commenting his Nature Genetics paper on mutations beneficial to patients with β-thalassemia and sickle cell anemia. The word organic here means that fetal hemoglobin production can be boosted without inserting foreign DNA.