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Gene Editing for Inherited Red Blood Cell Diseases

28 Mar 2022

Abstract excerpt

Today gene therapy is a real therapeutic option to address inherited hematological diseases that could be beneficial for thousands of patients worldwide. Currently, gene therapy is used to treat different monogenic hematological pathologies, including several red blood cell diseases such as β-thalassemia, sickle cell disease and pyruvate kinase deficiency. This approach is based on addition gene therapy, which...

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PMID: 35418876
DOI: 10.3389/fphys.2022.848261

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