Therapeutic efficacy in a hemophilia B model using a biosynthetic mRNA liver depot system

DNA-based gene therapy has considerable therapeutic potential, but the challenges associated with delivery continue to limit progress. Messenger RNA (mRNA) has the potential to provide for transient production of therapeutic proteins, without the need for nuclear delivery and without the risk of ins...

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Detalles Bibliográficos
Autores principales: DeRosa, F, Guild, B, Karve, S, Smith, L, Love, K, Dorkin, J R, Kauffman, K J, Zhang, J, Yahalom, B, Anderson, D G, Heartlein, M W
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2016
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5059749/
https://www.ncbi.nlm.nih.gov/pubmed/27356951
http://dx.doi.org/10.1038/gt.2016.46

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5059749/
https://www.ncbi.nlm.nih.gov/pubmed/27356951
http://dx.doi.org/10.1038/gt.2016.46

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