Posts tagged 'cancer'
The current universe of vaccines can be divided into two main categories, conventional vaccines and nucleic acid vaccines (Blakney et al.). Conventional vaccines are based on a protein adjuvant, and nucleic acid vaccines can be based on DNA or RNA. For this blog, we will focus on nucleic acid vaccines based on RNA structures and, in particular, self-amplifying RNA vaccines (Figure 1).
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. Before discussing these findings, the next section will briefly introduce the history of gene therapy and why IVT mRNA is attracting increasing interest in this context.
There is widespread interest in chemically modified mRNA (modRNA) synthesized by in vitro transcription (IVT) reactions, wherein one or more of the natural (aka wild-type) A, G, C, and U nucleotide 5’-triphosphates is replaced by base-modified analogs.