Engineered nucleic acids and methods of use thereof

摘要

Provided are compositions and methods for delivering biological moieties such as modified nucleic acids into cells to modulate protein expression. Such compositions and methods include the use of modified messenger RNAs, and are useful to treat or prevent diseases, disorders or conditions, or to improve a subject's heath or wellbeing.

主权项

1. A pharmaceutical formulation comprising:
i) an effective amount of a messenger ribonucleic acid (mRNA) sequence encoding an insulin polypeptide, wherein the mRNA sequence is at least 95% identical to the sequence of SEQ ID NO: 23; and ii) a pharmaceutically acceptable carrier,wherein the formulation is suitable for repeated intravenous administration to a mammalian subject in need thereof.