Article
Generation of an mESC model with a human hemophilia B nonsense mutation via CRISPR/Cas9 technology.
Stem cell research & therapy - 26 Jul 2022
Ma Yanchun, Sun Wenwen, Zhao Lidong, Yao Mingze, Wu Changxin, Su Pengfei, Yang Linhua, Wang Gang
Abstract excerpt
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. Although gene therapy has the potential to cure hemophilia, significant barriers are still needed to be overcome, e.g.,...
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