Low-dose AAV-CRISPR-mediated liver-specific knock-in restored hemostasis in neonatal hemophilia B mi...
Low-dose AAV-CRISPR-mediated liver-specific knock-in restored hemostasis in neonatal hemophilia B mice with subtle antibody response
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Publisher
London: Nature Publishing Group UK
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English
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Publisher
London: Nature Publishing Group UK
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AAV-delivered CRISPR/Cas9 (AAV-CRISPR) has shown promising potentials in preclinical models to efficiently insert therapeutic gene sequences in somatic tissues. However, the AAV input doses required were prohibitively high and posed serious risk of toxicity. Here, we performed AAV-CRISPR mediated homology-independent knock-in at a new target site i...
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Full title
Low-dose AAV-CRISPR-mediated liver-specific knock-in restored hemostasis in neonatal hemophilia B mice with subtle antibody response
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TN_cdi_doaj_primary_oai_doaj_org_article_8c34ac5c54144838ba20e5afe10957da
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_8c34ac5c54144838ba20e5afe10957da
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ISSN
2041-1723
E-ISSN
2041-1723
DOI
10.1038/s41467-022-34898-y
