Document Detail


NMR spectroscopy of aminoacylase 1 deficiency, a novel inborn error of metabolism.
MedLine Citation:
PMID:  17516490     Owner:  NLM     Status:  MEDLINE    
Abstract/OtherAbstract:
Aminoacylase 1 deficiency is a novel inborn error of metabolism. The clinical significance of the deficiency is under discussion, as well as the possible consequences of the defect for brain metabolism and function. This study includes the five originally published cases as well as three novel ones. NMR spectroscopy of urine, serum and cerebrospinal fluid has been used to study these patients. A typical profile with 11 accumulating N-acetylated amino acids was observed in urine from the patients. The concentration of most of the accumulating metabolites is typically 100-500 micromol/mmol creatinine. Two additional minor N-acetylated metabolites remain unidentified. The concentrations of the accumulating metabolites are <20 micromol/L in serum from the patients. Interestingly we found no evidence of an increased concentration of N-acetylated amino acids in the cerebrospinal fluid from one patient. Our data define aminoacylase 1 deficiency at the metabolite level providing a specific urinary profile of accumulating N-acetylated amino acids.
Authors:
Udo F H Engelke; Jörn Oliver Sass; Rudy N Van Coster; Erik Gerlo; Heike Olbrich; Stefan Krywawych; Jacqui Calvin; Claire Hart; Heymut Omran; Ron A Wevers
Publication Detail:
Type:  Journal Article    
Journal Detail:
Title:  NMR in biomedicine     Volume:  21     ISSN:  0952-3480     ISO Abbreviation:  NMR Biomed     Publication Date:  2008 Feb 
Date Detail:
Created Date:  2008-02-13     Completed Date:  2008-06-24     Revised Date:  -    
Medline Journal Info:
Nlm Unique ID:  8915233     Medline TA:  NMR Biomed     Country:  England    
Other Details:
Languages:  eng     Pagination:  138-47     Citation Subset:  IM    
Affiliation:
Radboud University Nijmegen Medical Center, Laboratory of Pediatrics and Neurology, Nijmegen, The Netherlands.
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MeSH Terms
Descriptor/Qualifier:
Acetylation
Adolescent
Amidohydrolases / deficiency*
Amino Acid Metabolism, Inborn Errors / enzymology*,  physiopathology*
Amino Acids / blood,  cerebrospinal fluid,  urine
Child, Preschool
Humans
Infant
Infant, Newborn
Magnetic Resonance Spectroscopy*
Male
Chemical
Reg. No./Substance:
0/Amino Acids; EC 3.5.-/Amidohydrolases; EC 3.5.1.14/aminoacylase I

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