Expression of therapeutic proteins after delivery of chemically modified mRNA in mice
Expression of therapeutic proteins after delivery of chemically modified mRNA in mice
M. Kormann,G. Hasenpusch,13 Authors,C. Rudolph
2011 · DOI: 10.1038/nbt.1733
Nature Biotechnology · 706 Citations
TLDR
In a mouse model of a lethal congenital lung disease caused by a lack of surfactant protein B (SP-B), twice weekly local application of an aerosol of modified SP-B mRNA to the lung restored 71% of the wild-type SP- B expression, and treated mice survived until the predetermined end of the study after 28 days.