In Vivo Outcome of Homology-Directed Repair at the HBB Gene in HSC Using Alternative Donor Template...
In Vivo Outcome of Homology-Directed Repair at the HBB Gene in HSC Using Alternative Donor Template Delivery Methods
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United States: Elsevier Inc
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Language
English
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United States: Elsevier Inc
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Gene editing following designer nuclease cleavage in the presence of a DNA donor template can revert mutations in disease-causing genes. For optimal benefit, reversion of the point mutation in HBB leading to sickle cell disease (SCD) would permit precise homology-directed repair (HDR) while concurrently limiting on-target non-homologous end joining...
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In Vivo Outcome of Homology-Directed Repair at the HBB Gene in HSC Using Alternative Donor Template Delivery Methods
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TN_cdi_doaj_primary_oai_doaj_org_article_5c5d0cc4529c44adb9273c8f4a9f26d9
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https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_5c5d0cc4529c44adb9273c8f4a9f26d9
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ISSN
2162-2531
E-ISSN
2162-2531
DOI
10.1016/j.omtn.2019.05.025
