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Treating Sickle Cell Disease: Gene Therapy Approaches

11 Sept 2024

Abstract excerpt

Sickle cell disease (SCD) is a hereditary blood disorder characterized by the presence of abnormal hemoglobin molecules and thus distortion (sickling) of the red blood cells. SCD causes chronic pain and organ damage and shortens life expectancy. Gene therapy emerges as a potentially curative approach for people with SCD who lack a matched sibling donor for hematopoietic stem cell transplantation. Here, we review...

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PMID: 39259977
DOI: 10.1146/annurev-pharmtox-022124-022000

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