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MRNA therapy for phenylketonuria

  • US 10,208,295 B2
  • Filed: 11/11/2016
  • Issued: 02/19/2019
  • Est. Priority Date: 10/22/2013
  • Status: Active Grant
First Claim
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1. A composition for treating phenylketonuria (PKU), comprising an mRNA encoding phenylalanine hydroxylase (PAH) at an effective dose amount encapsulated within a liposome,wherein the liposome comprises one or more cationic lipids, one or more non-cationic lipids, one or more cholesterol-based lipids and one or more PEG-modified lipids and wherein the liposome has a size less than 100 nm,wherein serum phenylalanine levels are reduced as compared to baseline serum phenylalanine levels before treatment,wherein the mRNA comprises SEQ ID NO:

  • 3.

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