mRNA FOR USE IN TREATMENT OF HUMAN GENETIC DISEASES
AssigneeTranslate Bio, Inc.
InventorsBraydon Charles Guild, Michael Heartlein, Frank DeRosa
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Abstract
Compositions for modulating the expression of a protein in a target cell comprising at least one RNA molecule which comprises at least one modification 5 conferring stability to the RNA, as well as related methods, are disclosed.
Claims20
A composition for modulating the expression of a protein in a target cell, wherein said composition comprises at least one RNA molecule and a transfer vehicle and wherein the RNA comprises at least one modification which confers stability to the RNA.
A method of treating a subject deficient in a protein, comprising administering a composition comprising an mRNA and a transfer vehicle, wherein the mRNA encodes a functional protein corresponding to the protein which is deficient in the subject, and wherein the mRNA comprises at least one modification which confers stability to the administered mRNA.
Dependencies are inferred from the claim text.