Lipid nanoparticle compositions and methods for mRNA delivery
First Claim
1. A method for delivery of messenger RNA (mRNA) for in vivo production of a secreted polypeptide, comprising administering, to a human, a composition comprising an mRNA that encodes the secreted polypeptide wherein the mRNA is encapsulated within a lipid nanoparticle, wherein the administering of the composition results in expression of the secreted polypeptide encoded by the mRNA that is detectable in serum at least 72 hours after administration, and wherein the lipid nanoparticle comprises one or more PEG-modified lipids.
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Abstract
Disclosed herein are compositions and methods for modulating the production of a protein in a target cell. The compositions and methods disclosed herein are capable of ameliorating diseases associated with protein or enzyme deficiencies.
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Citations
22 Claims
- 1. A method for delivery of messenger RNA (mRNA) for in vivo production of a secreted polypeptide, comprising administering, to a human, a composition comprising an mRNA that encodes the secreted polypeptide wherein the mRNA is encapsulated within a lipid nanoparticle, wherein the administering of the composition results in expression of the secreted polypeptide encoded by the mRNA that is detectable in serum at least 72 hours after administration, and wherein the lipid nanoparticle comprises one or more PEG-modified lipids.
Specification