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CRISPR/Cas9-mediated genome editing via postnatal administration of AAV vector cures haemophilia B mice
Haemophilia B, a congenital haemorrhagic disease caused by mutations in coagulation factor IX gene (F9), is considered an appropriate target for genome editing technology. Here, we describe treatment strategies for haemophilia B mice using the clustered regularly interspaced short palindromic repeat...
| Autores principales: | , , , , , , , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
Nature Publishing Group UK
2017
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5482879/ https://www.ncbi.nlm.nih.gov/pubmed/28646206 http://dx.doi.org/10.1038/s41598-017-04625-5 |