Hemophilia A ameliorated in mice by CRISPR-based in vivo genome editing of human Factor VIII
Hemophilia A ameliorated in mice by CRISPR-based in vivo genome editing of human Factor VIII
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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
Hemophilia A is a monogenic disease with a blood clotting factor VIII (FVIII) deficiency caused by mutation in the factor VIII (
F8)
gene. Current and emerging treatments such as FVIII protein injection and gene therapies via AAV-delivered
F8
transgene in an episome are costly and nonpermanent. Here, we describe a CRISPR/Cas9-based
i...
Alternative Titles
Full title
Hemophilia A ameliorated in mice by CRISPR-based in vivo genome editing of human Factor VIII
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TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_6856096
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_6856096
Other Identifiers
ISSN
2045-2322
E-ISSN
2045-2322
DOI
10.1038/s41598-019-53198-y
