Article

Cas9-AAV6 Gene Correction of Beta-Globin in Autologous HSCs Improves Sickle Cell Disease Erythropoiesis in Mice

2020-10-13

Abstract excerpt

CRISPR/Cas9-mediated beta-globin ( HBB ) gene correction of Sickle Cell Disease (SCD) patient-derived hematopoietic stem cells (HSCs) in combination with autologous transplantation represents a novel paradigm in gene therapy. Although several Cas9-based HBB -correction approaches have been proposed, functional correction of in vivo erythropoiesis has not been investigated. Here, we used a humanized globin-cluste...

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Literature Corpus work: ccb1000b-d73f-5876-8ccb-8d6f308bbfb5
DOI: 10.1101/2020.10.13.338319

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