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

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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Bibliographic Details
Main Authors: DeRosa, F (Author), Guild, B (Author), Karve, S (Author), Smith, L (Author), Zhang, J (Author), Yahalom, B (Author), Heartlein, M W (Author), Love, Kerry R. (Contributor), Dorkin, Joseph Robert (Contributor), Kauffman, Kevin John (Contributor), Anderson, Daniel Griffith (Contributor)
Other Authors: Massachusetts Institute of Technology. Department of Chemical Engineering (Contributor), Koch Institute for Integrative Cancer Research at MIT (Contributor)
Format: Article
Language:English
Published: Nature Publishing Group, 2017-04-20T19:12:19Z.
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