Is a
Patent attributes
Patent Applicant
Current Assignee
Patent Jurisdiction
Patent Number
Date of Patent
December 20, 2016
Patent Application Number
14521323
Date Filed
October 22, 2014
Patent Citations Received
...
Patent Primary Examiner
Patent abstract
The present invention provides, among other things, methods of treating phenylketonuria (PKU), including administering to a subject in need of treatment a composition comprising an mRNA encoding phenylalanine hydroxylase (PAH) at an effective dose and an administration interval such that at least one symptom or feature of PKU is reduced in intensity, severity, or frequency or has delayed in onset. In some embodiments, the mRNA 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.
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