532. Optimization of Lentivirus Vector RNA Polymerase II Driven microRNA Embedded shRNAs for Enhanced Processing and Efficient Knockdown of Bcl11a for Induction of Fetal Hemoglobin in Erythroid Cells

Molecular Therapy - United States
doi 10.1016/s1525-0016(16)35545-9
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Elsevier BV

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