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, Guild, B, Karve, S, Smith, L, Zhang, J, Yahalom, B, Heartlein, M W, Love, Kerry R., Dorkin, Joseph Robert, Kauffman, Kevin John, Anderson, Daniel Griffith
Other Authors: Massachusetts Institute of Technology. Department of Chemical Engineering
Format: Article
Language:en_US
Published: Nature Publishing Group 2017
Online Access:http://hdl.handle.net/1721.1/108314
https://orcid.org/0000-0002-9436-2453
https://orcid.org/0000-0001-5629-4798

Internet

http://hdl.handle.net/1721.1/108314
https://orcid.org/0000-0002-9436-2453
https://orcid.org/0000-0001-5629-4798

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