Targeted gene correction of human hematopoietic stem cells for the treatment of Wiskott - Aldrich Sy...
Targeted gene correction of human hematopoietic stem cells for the treatment of Wiskott - Aldrich Syndrome
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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
Wiskott-Aldrich syndrome (WAS) is an X-linked primary immunodeficiency with severe platelet abnormalities and complex immunodeficiency. Although clinical gene therapy approaches using lentiviral vectors have produced encouraging results, full immune and platelet reconstitution is not always achieved. Here we show that a CRISPR/Cas9-based genome edi...
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Full title
Targeted gene correction of human hematopoietic stem cells for the treatment of Wiskott - Aldrich Syndrome
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TN_cdi_doaj_primary_oai_doaj_org_article_e93094c9c51f4c1aa746e611fd6ea64c
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https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_e93094c9c51f4c1aa746e611fd6ea64c
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ISSN
2041-1723
E-ISSN
2041-1723
DOI
10.1038/s41467-020-17626-2
