Editing a γ-globin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease phenotype

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Shared by NobleBlocks on Feb 14, 2020 • 12:00 AM UTC

Authors:

Leslie Weber

Giacomo Frati

Tristan Felix

Abstract

Editing the fetal γ-globin promoters in hematopoietic stem cells from sickle cell disease patients induces therapeutic γ-globin levels.

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