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Gene therapies for transfusion dependent β‐thalassemia: Current status and critical criteria for success

20 Jun 2020

Abstract excerpt

Thalassemia is one of the most prevalent monogenic diseases usually caused by quantitative defects in the production of β-globin leading to severe anemia. Technological advances in genome sequencing, stem cell selection, viral vector development, transduction and gene editing strategies now allow for efficient exvivo genetic manipulation of human stem cells that can lead to production of hemoglobin, leading to a...

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PMID: 32562290
DOI: 10.1002/ajh.25909

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