Partial rescue of neuropathology in the murine model of PKU following administration of recombinant phenylalanine ammonia lyase (pegvaliase)
| Autor: | Marc H. Goldfinger, William L. Zeile, Laurie S. Tsuruda, Jonathan D. Cooper, Carley R. Corado, Charles A. O'Neill, Philip J. Laipis |
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| Rok vydání: | 2017 |
| Předmět: |
0301 basic medicine
congenital hereditary and neonatal diseases and abnormalities medicine.medical_specialty Tyrosine 3-Monooxygenase Endocrinology Diabetes and Metabolism Phenylalanine Phenylalanine ammonia-lyase Neuropathology Placebo Biochemistry law.invention 03 medical and health sciences Mice 0302 clinical medicine Endocrinology law Internal medicine Phenylketonurias Genetics medicine Phenylketonuria Animals Humans Molecular Biology Phenylalanine Ammonia-Lyase chemistry.chemical_classification Neurotransmitter Agents Tyrosine hydroxylase nutritional and metabolic diseases Brain Recombinant Proteins Disease Models Animal 030104 developmental biology Enzyme Monoamine neurotransmitter chemistry Recombinant DNA Recombinant phenylalanine ammonia lyase 030217 neurology & neurosurgery |
| Zdroj: | Goldfinger, M, Zeile, W L, Corado, C R, O'Neill, C A, Tsuruda, L S, Laipis, P J & Cooper, J D 2017, ' Partial rescue of neuropathology in the murine model of PKU following administration of recombinant phenylalanine ammonia lyase (pegvaliase) ', MOLECULAR GENETICS AND METABOLISM . https://doi.org/10.1016/j.ymgme.2017.04.013 |
| ISSN: | 1096-7206 |
| DOI: | 10.1016/j.ymgme.2017.04.013 |
| Popis: | 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. |
| Databáze: | OpenAIRE |
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