Partial rescue of neuropathology in the murine model of PKU following administration of recombinant phenylalanine ammonia lyase (pegvaliase)

Marc Goldfinger, William L. Zeile, Carley R. Corado, Charles A. O'Neill, Laurie S. Tsuruda, Philip J. Laipis, Jonathan D. Cooper

Research output: Contribution to journalArticle

3 Scopus citations

Abstract

Pegylated recombinant phenylalanine ammonia lyase (pegvaliase) is an enzyme substitution therapy being evaluated for the treatment of phenylketonuria (PKU). PKU is characterized by elevated plasma phenylalanine, which is thought to lead to a deficiency in monoamine neurotransmitters and ultimately, neurocognitive dysfunction. A natural history evaluation in a mouse model of PKU demonstrated a profound decrease in tyrosine hydroxylase (TH) immunoreactivity in several brain regions, beginning at 4 weeks of age. Following treatment with pegvaliase, the number of TH positive neurons was increased in several brain regions compared to placebo treated ENU2 mice.

Original languageEnglish
Pages (from-to)33-35
Number of pages3
JournalMolecular genetics and metabolism
Volume122
Issue number1-2
DOIs
StatePublished - Sep 2017
Externally publishedYes

Keywords

  • Phenylketonuria
  • Recombinant phenylalanine ammonia lyase
  • Tyrosine hydroxylase

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