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Cell and Gene Therapy for Anemia: Hematopoietic Stem Cells and Gene Editing

10 Jun 2021

Abstract excerpt

Hereditary anemia has various manifestations, such as sickle cell disease (SCD), Fanconi anemia, glucose-6-phosphate dehydrogenase deficiency (G6PDD), and thalassemia. The available management strategies for these disorders are still unsatisfactory and do not eliminate the main causes. As genetic aberrations are the main causes of all forms of hereditary anemia, the optimal approach involves repairing the...

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PMID: 34200975
DOI: 10.3390/ijms22126275

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