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Article

A Review of Gene Therapies for Hemoglobinopathies

3 May 2024

Abstract excerpt

Due to the significant morbidity and mortality of hemoglobinopathies, curative options have long been pursued. The overall goal of gene therapy is to modify a patient’s own hematopoietic stem cells to overcome the deleterious effects of the underlying genetic defect by gene addition, gene editing, or gene silencing. Gene addition incorporates genes with superior function than the abnormal gene; gene editing takes...

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PMID: 39145521
DOI: 10.1080/03630269.2024.2369534

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