Gene Therapy and Gene Editing for β-Thalassemia
Georgios E. Christakopoulos, Rahul Telange, Jonathan Yen, Mitchell J. Weiss
Topics & Concepts
Genetic enhancementThalassemiaGenome editingHaematopoiesisGeneFetal hemoglobinTransduction (biophysics)Stem cellMedicineGenomeBioinformaticsBiologyComputational biologyGeneticsFetusPregnancyBiochemistryCRISPR and Genetic EngineeringHemoglobinopathies and Related DisordersPrenatal Screening and Diagnostics