| abstract |
Composition comprising a) at least one mRNA molecule where at least part thereof encodes a functional secreted polypeptide (preferably erythropoietin or a-functional galactosidase); and b) a transfer vehicle comprising a lipid nanoparticle (less than 100 nm in size) comprising one or more cationic lipids, one or more non-cationic lipids, one or more cholesterol-based lipids, and one or more lipids modified by PEG Preferably the mRNA molecule comprises a modification that increases its stability (preferably the inclusion of a Cap1 structure or a poly A end). In one embodiment the composition is in lyophilized form (reconstituted or not), and may comprise an agent that facilitates the transfer of the mRNA molecule to a target cell. Use of a composition comprising a) at least one mRNA encoding a functional secreted polypeptide (preferably those listed in R18, and with the addition of CAP at the 5 'end and a polyA tract at the 3' end), and b) a transfer vehicle comprising a lipid nanoparticle, for the preparation of a medicament useful in the treatment of a deficiency in a functional polypeptide. Method for producing a functional secreted polypeptide in a target cell that comprises administering the claimed composition, where after administration thereof, the mRNA is translated into a target cell in order to produce the functional polypeptide. |