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US 9,956,271 B2Granted
mRNA for use in treatment of human genetic diseases
AssigneeTranslate Bio, 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.
Claims9
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 ornithine transcarbamylase (OTC), wherein the urea cycle disorder is OTC deficiency and
- wherein the mRNA comprises a 5′ untranslated region (UTR) comprising SEQ ID NO:
- 1, wherein SEQ ID NO:
- 1 enhances the expression of the enzyme.
2 more elements
Dependencies are inferred from the claim text.