A systematic review of clinical trials for gene therapies for β-hemoglobinopathy around the world

Felipe Augusto Rós,Samuel Campanelli Freitas Couto,Jonathan Milhomens,Ian Ovider, Karina Tozatto Maio,Viviane Jennifer,Rodrigo Nalio Ramos,Virginia Picanço-Castro,Simone Kashima,Rodrigo T. Calado,Luciana Rodrigues Carvalho Barros,Vanderson Rocha

Cytotherapy（2023）

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摘要

Geographic trial concentration indicates the high costs of this technology, logistical issues and social challenges that need to be overcome for gene therapy to reach low- and middle-income countries where SCD and TDT are prevalent and where they most impact the patient's health.

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关键词

beta-thalassemia,clinical trials,gene therapy,sickle cell disease,systematic review

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