Generation of an mESC model with a human hemophilia B nonsense mutation via CRISPR/Cas9 technology

Generation of an mESC model with a human hemophilia B nonsense mutation via CRISPR/Cas9 technology

Abstract Background Hemophilia B is a rare inherited genetic bleeding disorder caused by a deficiency or lack of coagulation factor IX, the gene for which (F9) is located on the X chromosome. Hemophilia B is currently incurable and the standard treatment is coagulation factor replacement therapy. Al...

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Bibliographic Details
Main Authors: Yanchun Ma, Wenwen Sun, Lidong Zhao, Mingze Yao, Changxin Wu, Pengfei Su, Linhua Yang, Gang Wang
Format: Article
Language:English
Published: BMC 2022-07-01
Series:Stem Cell Research & Therapy
Subjects:
Hemophilia
Mouse embryonic stem cell
Nonsense mutation
CRISPR/Cas9 technology
Hepatocyte-like cell
Online Access:https://doi.org/10.1186/s13287-022-03036-2

Internet

https://doi.org/10.1186/s13287-022-03036-2

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