Modified polynucleotides for the production of biologics and proteins associated with human disease

摘要

The invention relates to compositions and methods for the preparation, manufacture and therapeutic use of polynucleotides, primary transcripts and mmRNA molecules.

主权项

1. An isolated polynucleotide comprising;
(a) a first region of linked nucleosides, said first region encoding a polypeptide of interest, said polypeptide of interest is SEQ ID NO: 1089; (b) a first flanking region located at the 5′ terminus of said first region comprising at least one 5′ terminal cap; (c) a second flanking region located at the 3′ terminus of said first region comprising a 3′ tailing sequence of linked nucleosides; andwherein at least one of said linked nucleosides of (a) comprises at least one modification as compared to the chemical structure of an A, G, U or C ribonucleoside.