Curative therapy for hemoglobinopathies: an International Society for Cell & Gene Therapy Stem Cell Engineering Committee review comparing outcomes, accessibility and cost of ex vivo stem cell gene therapy versus allogeneic hematopoietic stem cell transplantation
Alexis Leonard, Alice Bertaina, Carmem Bonfim, Sandra Cohen, Susan E. Prockop, Duncan Purtill, Athena Russell, Jaap Jan Boelens, Robert Wynn, Annalisa Ruggeri, Allistair Abraham
Topics & Concepts
MedicineGenetic enhancementThalassemiaHematopoietic stem cell transplantationStem cellDiseaseHematopoietic stem cellTransplantationCell therapyStem-cell therapyHaematopoiesisIntensive care medicineImmunologyOncologyInternal medicineGeneBiologyGeneticsBiochemistryHemoglobinopathies and Related DisordersPrenatal Screening and DiagnosticsParvovirus B19 Infection Studies