Cas9-AAV6 gene correction of beta-globin in autologous HSCs improves sickle cell disease erythropoie...
Cas9-AAV6 gene correction of beta-globin in autologous HSCs improves sickle cell disease erythropoiesis in mice
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Publisher
London: Nature Publishing Group UK
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Language
English
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Publisher
London: Nature Publishing Group UK
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Contents
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 recent paradigm in gene therapy. Although several Cas9-based
HBB
-correction approaches have been proposed, functional correction of in vivo ery...
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Full title
Cas9-AAV6 gene correction of beta-globin in autologous HSCs improves sickle cell disease erythropoiesis in mice
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TN_cdi_doaj_primary_oai_doaj_org_article_33cf7e3f25534b81a1ff9bc124c467e5
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https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_33cf7e3f25534b81a1ff9bc124c467e5
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ISSN
2041-1723
E-ISSN
2041-1723
DOI
10.1038/s41467-021-20909-x
