| abstract |
Disclosed are compositions and methods for modulating protein production in target cells. The compositions and methods disclosed herein can ameliorate diseases associated with protein or enzyme deficiencies. For example, the present invention provides a composition comprising (a) at least one mRNA molecule that at least partially encodes a functional secreted polypeptide, and (b) a transport vehicle comprising lipid nanoparticles. The present invention is also a method of treating a subject deficient in a functional polypeptide, comprising (a) at least one mRNA that at least partially encodes said functional secreted polypeptide, and (b) a lipid. Administering a composition comprising a transport vehicle comprising nanoparticles, and after administration of said composition, said mRNA is expressed in a target cell to produce said functionally secreted polypeptide. Also provide. [Selection figure] None |