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

Molecular therapy : the journal of the American Society of Gene Therapy

Sean Mc Cafferty, Joyca De Temmerman, Tasuku Kitada, …, Siegrid De Baere, Francis Combes, Niek N Sanders

• DOI: 10.1016/j.ymthe.2020.11.010
• PMID: 33186690
• PMCID: PMC7934578