In Vivo Validation of a Reversible Small Molecule-Based Switch for Synthetic Self-Amplifying mRNA Regulation

Synthetic mRNA therapeutics have the potential to revolutionize healthcare, as they enable patients to produce therapeutic proteins inside their own bodies. However, convenient methods that allow external control over the timing and magnitude of protein production after in vivo delivery of synthetic...

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Bibliografiske detaljer
Udgivet i:Mol Ther
Main Authors: Mc Cafferty, Sean, De Temmerman, Joyca, Kitada, Tasuku, Becraft, Jacob R., Weiss, Ron, Irvine, Darrell J., Devreese, Mathias, De Baere, Siegrid, Combes, Francis, Sanders, Niek N.
Format: Artigo
Sprog:Inglês
Udgivet: American Society of Gene & Cell Therapy 2021
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Original Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7934578/
https://ncbi.nlm.nih.gov/pubmed/33186690
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2020.11.010
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