PHARMACEUTICAL COMPOSITION CONTAINING A STABILISED mRNA OPTIMISED FOR TRANSLATION IN ITS CODING REGIONS

摘要

The present invention relates to a pharmaceutical composition comprising a modified mRNA that is stabilised by sequence modifications and optimised for translation. The pharmaceutical composition according to the invention is particularly well suited for use as an inoculating agent, as well as a therapeutic agent for tissue regeneration. In addition, a process is described for determining sequence modifications that promote stabilisation and translational efficiency of modified mRNA of the invention.

主权项

1. A method of providing insulin expression in a subject comprising administering an effective amount of a pharmaceutical composition comprising a mRNA that encodes insulin to the subject, wherein the mRNA that encodes insulin comprises:
(i) an increased G/C content relative to a wild type RNA encoding for insulin; or (ii) at least one codon of a wild-type sequence recognized by a rare cellular tRNA is replaced with a codon recognized by an abundant cellular tRNA, and wherein said rare cellular tRNA and said abundant cellular tRNA recognize the same amino acid.