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Hematopoietic Stem Cell Gene-Addition/Editing Therapy in Sickle Cell Disease

4 Jun 2022

Abstract excerpt

Autologous hematopoietic stem cell (HSC)-targeted gene therapy provides a one-time cure for various genetic diseases including sickle cell disease (SCD) and β-thalassemia. SCD is caused by a point mutation (20A > T) in the β-globin gene. Since SCD is the most common single-gene disorder, curing SCD is a primary goal in HSC gene therapy. β-thalassemia results from either the absence or the reduction of β-globin...

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PMID: 35681538
DOI: 10.3390/cells11111843

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