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Article

Preclinical Evaluation of a Novel Lentiviral Vector Driving Lineage-Specific BCL11A Knockdown for Sickle Cell Gene Therapy

17 Mar 2020

Abstract excerpt

In this work we provide preclinical data to support initiation of a first-in-human trial for sickle cell disease (SCD) using an approach that relies on reversal of the developmental fetal-to-adult hemoglobin switch. Erythroid-specific knockdown of BCL11A via a lentiviral-encoded microRNA-adapted short hairpin RNA (shRNA miR ) leads to reactivation of the gamma-globin gene while simultaneously reducing expression...

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PMID: 32300607
DOI: 10.1016/j.omtm.2020.03.015

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