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

Research output: Contribution to journalArticle

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

Original languageEnglish
JournalMOLECULAR GENETICS AND METABOLISM
Early online date29 Apr 2017
DOIs
Accepted/In press27 Apr 2017
E-pub ahead of print29 Apr 2017

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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.

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