CV Lattes
ORCID
Abstract
Sickle cell disease (SCD) is a severe disease caused by a single mutation in the beta-globin gene (HBB). The only curative therapy for SCD is allogeneic hematopoietic stem cell transplantation (alloHSCT), however most patients do not have a suitable donor. Gene therapy is a promising curative treatment for SCD. However, which is the best genome editing strategy remains an open question. A…