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US 2016/0101160 A1Published application
mRNA FOR USE IN TREATMENT OF HUMAN GENETIC DISEASES
AssigneeShire Human Genetic Therapies, Inc.
InventorsBraydon Charles Guild, Michael Heartlein, Frank DeRosa
Life of this patent
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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.
Claims13
1-20. (canceled)
A method of treating a urea cycle disorder comprising
- administering to a subject suffering from or susceptible to a urea cycle disorder, a composition comprising an mRNA encoding an enzyme, encapsulated within a liposome;
- wherein the enzyme is selected from ornithine transcarbamylase (OTC), carbamyl phosphate synthetase (CPS), argininosuccinate synthetase 1 (ASS1) argininosuccinate lyase (ASL), or arginase (ARG).
Dependencies are inferred from the claim text.