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Autologous and Heterologous Cell Therapy for Hemophilia B toward Functional Restoration of Factor IX
Hemophilia B is an ideal target for gene- and cell-based therapies because of its monogenic nature and broad therapeutic index. Here, we demonstrate the use of cell therapy as a potential long-term cure for hemophilia B in our FIX-deficient mouse model. We show that transplanted, cryopreserved, cada...
| Autores principales: | , , , , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
2018
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5987250/ https://www.ncbi.nlm.nih.gov/pubmed/29719266 http://dx.doi.org/10.1016/j.celrep.2018.03.121 |