Coordinated β-globin expression and α2-globin reduction in a multiplex lentiviral gene therapy vector for β-thalassemia

出版年份 2021 全文链接

标题

作者

关键词

gene therapy, thalassemias, lentiviral vector, shRNAmir, globin, BB305, LVβ-shα2, RNA inteference, insertional mutagenesis, hemoglobin disorder

出版物

MOLECULAR THERAPY

Volume -, Issue -, Pages -

出版商

Elsevier BV

发表日期

2021-05-01

DOI

10.1016/j.ymthe.2021.04.037

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