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

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 g...

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Detalles Bibliográficos
Autores principales: Ma, Yanchun, Sun, Wenwen, Zhao, Lidong, Yao, Mingze, Wu, Changxin, Su, Pengfei, Yang, Linhua, Wang, Gang
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9327398/
https://www.ncbi.nlm.nih.gov/pubmed/35883203
http://dx.doi.org/10.1186/s13287-022-03036-2

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