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

Published 2021 View Full Article

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Title
Coordinated β-globin expression and α2-globin reduction in a multiplex lentiviral gene therapy vector for β-thalassemia
Authors
Keywords
gene therapy, thalassemias, lentiviral vector, shRNAmir, globin, BB305, LVβ-shα2, RNA inteference, insertional mutagenesis, hemoglobin disorder
Journal
MOLECULAR THERAPY
Volume -, Issue -, Pages -
Publisher
Elsevier BV
Online
2021-05-01
DOI
10.1016/j.ymthe.2021.04.037

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