Litcius/Paper detail

A new frontier: FDA approvals for gene therapy in sickle cell disease

Alexis Leonard, John F. Tisdale

2024Molecular Therapy71 citationsDOIOpen Access PDF

Topics & Concepts

Genetic enhancementDiseaseDrug approvalMedicineGeneBiologyPharmacologyGeneticsInternal medicineDrugCRISPR and Genetic EngineeringHemoglobinopathies and Related DisordersIron Metabolism and Disorders