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US 8,853,377 B2Granted
mRNA for use in treatment of human genetic diseases
AssigneeShire Human Genetic Therapies, Inc.
InventorsBraydon Charles Guild, Michael Heartlein
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 conferring stability to the RNA, as well as related methods, are disclosed.
Claims14
A composition for delivery of mRNA for protein expression in a target cell, comprising at least one mRNA molecule comprising a sequence encoding a protein of interest and a 5′ untranslated region (UTR) comprising SEQ ID NO:
- 1, wherein SEQ ID NO:
- 1 enhances the expression of the protein of interest.
Dependencies are inferred from the claim text.