- Systemic delivery of factor IX messenger RNA for protein replacement therapy.
Systemic delivery of factor IX messenger RNA for protein replacement therapy.
Proceedings of the National Academy of Sciences of the United States of America (2017-02-17)
Suvasini Ramaswamy, Nina Tonnu, Kiyoshi Tachikawa, Pattraranee Limphong, Jerel B Vega, Priya P Karmali, Pad Chivukula, Inder M Verma
PMID28202722
RÉSUMÉ
Safe and efficient delivery of messenger RNAs for protein replacement therapies offers great promise but remains challenging. In this report, we demonstrate systemic, in vivo, nonviral mRNA delivery through lipid nanoparticles (LNPs) to treat a Factor IX (
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