HSCT remains the only cure for patients with transfusion-dependent thalassemia until gene therapy strategies are proven to be safe
Christina Oikonomopoulou, Evgenios Goussetis
Topics & Concepts
MedicineGenetic enhancementThalassemiaHematopoietic stem cell transplantationIntensive care medicineTransplantationSiblingClinical trialChelation therapyHuman leukocyte antigenPediatricsImmunologyInternal medicineGeneBiochemistryChemistryAntigenPsychologyDevelopmental psychologyHemoglobinopathies and Related DisordersCRISPR and Genetic EngineeringParvovirus B19 Infection Studies