Successful correction of hemophilia by CRISPR/Cas9 genome editing in vivo: delivery vector and immune responses are the key to success
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...
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| Main Authors: | , |
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| Format: | Article |
| Language: | English |
| Published: |
Springer Nature
2016-04-01
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| Series: | EMBO Molecular Medicine |
| Online Access: | https://doi.org/10.15252/emmm.201606325 |
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