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Gene Therapy of the β Hemoglobinopathies: Success and Challenges

Emmanuel Payen,Philippe Leboulch

Hemoglobin（2019）

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Abstract
The very large number of known mutations responsible for the β-thalassemias, renders gene therapy by gene addition ideally suited for regulatory product approval rather than the many mutation-speci...
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Key words
hemoglobinopathies,gene therapy
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