Abstract
Hemophilia B is a serious hemostasis disorder due to mutations of the factor IX gene in the X chromosome. Gene therapy has gained momentum in recent years as a therapeutic option for hemophilia B. In hemophilia, reconstitution with a mere 1–2% of the clotting factor improves the quality of life, while 5–20% suffices to ameliorate the bleeding disorder. A paper by Guan et al (2016) in this issue of EMBO Molecular Medicine reports on the direct CRISPRs/Cas9‐mediated correction in the liver of a hemophilia‐causing point mutation in FIX.
Publication types
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Research Support, Non-U.S. Gov't
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Comment
MeSH terms
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CRISPR-Cas Systems*
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Clustered Regularly Interspaced Short Palindromic Repeats
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Gene Editing*
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Genome
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Hemophilia A / genetics
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Humans