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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Bibliografski detalji
Izdano u:Gene Ther
Glavni autori: 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
Format: Artigo
Jezik:Inglês
Izdano: Nature Publishing Group 2016
Teme:
Original Article
Online pristup:https://ncbi.nlm.nih.gov/pmc/articles/PMC5059749/
https://ncbi.nlm.nih.gov/pubmed/27356951
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/gt.2016.46
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