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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...
Autores principales: | , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
BioMed Central
2022
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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 |