The Potential of Flexible Lipid Nanoparticle Formulations for mRNA Delivery in Preclinical Studies
Messenger RNA (mRNA) is an attractive therapeutic agent because it can deliver gene-replacement therapies for inherited metabolic diseases. Lipid nanoparticle-mRNA vaccines have already entered clinical trials to treat ornithine transcarbamylase deficiency, methylmalonic acidaemia and propionic acidaemia12. Flexibility Lipid nanoparticles are a versatile, robust, non-viral platform for delivering genetic material to cells. They have the potential …