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...

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Detalles Bibliográficos
Autores principales: Ramaswamy, Suvasini, Tonnu, Nina, Menon, Tushar, Lewis, Benjamin M., Green, Kevin T., Wampler, Derek, Monahan, Paul E., Verma, Inder M.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: 2018
Materias:
Article
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

Internet

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

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