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Gene Editing

We offer a variety of gene editing tools including Cas9, Cas9 nickase and mRNAs, as well as Cre recombinases for traditional recombination. View all!
“minRNA”—mRNAs with minimalistic untranslated regions (UTRs) are highly functional In Vitro and In Vivo

Introduction Synthetic mRNA produced by in vitro transcription (IVT) is the active pharmaceutical ingredient of approved vaccines and of many drugs under development. IVT mRNA typically contains several hundred bases of non-coding untranslated regions (UTRs) that are involved in the stabilization and translation of the mRNA. Remarkably, new research has discovered that these UTRs can be virtually eliminated without compromising translation of the encoded protein of interest. ...
TL Optimizing the Performance of IVT mRNA Using N1-Methylpseudouridine (N1mΨ)—Part 2: Heart Gene Therapy

Posted in the Zone on May 25, 2021 , part 1 of this series on optimizing the performance of in vitro translated (IVT) mRNA using N1-methylpseudouridine (N1mΨ) provided a historical introduction to the development of this chemically modified base. Part 1 focused on a 2015 study and an initial 2018 in vivo optimization study of N1mΨ-mRNA. This Part 2 blog features a subsequent 2019 publication on in vitro optimization of N1mΨ-mRNA for future heart gene therapy. Befor...