Lipid formulations for delivery of messenger RNA
First Claim
1. A method of treating cystic fibrosis comprisingadministering to subject in need of treatment a composition comprising an mRNA encoding a cystic fibrosis transmembrane conductance regulator (CFTR) protein encapsulated within a liposome such that the administering of the composition results in the expression of the CFTR protein encoded by the mRNA in one or more tissues affected by cystic fibrosis;
- wherein the liposome comprises a cationic lipid of formula I-c;
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Abstract
The present invention provides, among other things, methods of delivering mRNA in vivo, including administering to a subject in need of delivery a composition comprising an mRNA encoding a protein, encapsulated within a liposome such that the administering of the composition results in the expression of the protein encoded by the mRNA in vivo, wherein the liposome comprises a cationic lipid of formula I-c:
or a pharmaceutically acceptable salt thereof.
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Citations
9 Claims
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1. A method of treating cystic fibrosis comprising
administering to subject in need of treatment a composition comprising an mRNA encoding a cystic fibrosis transmembrane conductance regulator (CFTR) protein encapsulated within a liposome such that the administering of the composition results in the expression of the CFTR protein encoded by the mRNA in one or more tissues affected by cystic fibrosis; wherein the liposome comprises a cationic lipid of formula I-c; - View Dependent Claims (2, 3, 4, 5, 6, 7, 8, 9)
Specification