Article

Restoration of β-globin expression with optimally designed lentiviral vector for β-thalassemia treatment in Chinese patients

2020-07-21

Abstract excerpt

β-thalassemia is one of the most prevalent genetic diseases worldwide. The current treatment for β–thalassemia is allogeneic hematopoietic stem cell transplantation (HSCT), which is limited due to lack of matched donors. Gene therapy has been developed as an alternative therapeutic option for transfusion-ependent β -thalassemia (TDT). However, successful gene therapy for β -thalassemia patients in China has not...

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Literature Corpus work: e12c7a62-9931-5a3c-b6a0-51f875e0e530
DOI: 10.1101/2020.07.18.209759

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