Pulmonary delivery of mRNA
First Claim
Patent Images
1. A method of delivery of messenger RNA (mRNA) for in vivo expression of protein, comprisingadministering via inhalation from a nebulizer to a subject a composition comprising an mRNA encoding full-length exogenous CFTR protein such that the administering of the composition results in in vivo expression of the full-length exogenous CFTR protein encoded by the mRNA in lung cells of the subject, wherein the composition further comprises polyethylenimine (PEI).
3 Assignments
0 Petitions
Accused Products
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.
372 Citations
6 Claims
-
1. A method of delivery of messenger RNA (mRNA) for in vivo expression of protein, comprising
administering via inhalation from a nebulizer to a subject a composition comprising an mRNA encoding full-length exogenous CFTR protein such that the administering of the composition results in in vivo expression of the full-length exogenous CFTR protein encoded by the mRNA in lung cells of the subject, wherein the composition further comprises polyethylenimine (PEI).
-
4. A method of delivery of messenger RNA (mRNA) for in vivo expression of protein, comprising
administering via inhalation from a nebulizer to a subject a composition comprising an mRNA encoding full-length exogenous CFTR protein such that the administering of the composition results in in vivo expression of the full-length exogenous CFTR protein encoded by the mRNA in lung cells of the subject, wherein the mRNA encoding the full-length exogenous CFTR protein is encapsulated in a liposome comprising one or more cationic lipids, one or more non-cationic lipids, one or more cholesterol-based lipids and one or more PEG-modified lipids.
Specification