Gene therapy using mRNA is facing challenges concerning delivery, immunogenicity, translation efficiency, and production costs of therapeutic mRNA. Chemical modification of mRNA could address all of these issues. Herein we present the discovery of new 5’-mRNA caps and the development of chemical methods to install these caps on mRNA. We disclose methods for modification of the 3’-end of mRNA and show that both 5’- and 3’-modification beneficially impacts stability and expression levels of mRNAs in biochemical assays and cellular settings.

Chemical modification of mRNA: Toward enabling gene therapy

254th National Meeting (2017)

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