Document Detail


Loss of dermatan-4-sulfotransferase 1 function results in adducted thumb-clubfoot syndrome.
MedLine Citation:
PMID:  20004762     Owner:  NLM     Status:  MEDLINE    
Abstract/OtherAbstract:
Adducted thumb-clubfoot syndrome is an autosomal-recessive disorder characterized by typical facial appearance, wasted build, thin and translucent skin, congenital contractures of thumbs and feet, joint instability, facial clefting, and coagulopathy, as well as heart, kidney, or intestinal defects. We elucidated the molecular basis of the disease by using a SNP array-based genome-wide linkage approach that identified distinct homozygous nonsense and missense mutations in CHST14 in each of four consanguineous families with this disease. The CHST14 gene encodes N-acetylgalactosamine 4-O-sulfotransferase 1 (D4ST1), which catalyzes 4-O sulfation of N-acetylgalactosamine in the repeating iduronic acid-alpha1,3-N-acetylgalactosamine disaccharide sequence to form dermatan sulfate. Mass spectrometry of glycosaminoglycans from a patient's fibroblasts revealed absence of dermatan sulfate and excess of chondroitin sulfate, showing that 4-O sulfation by CHST14 is essential for dermatan sulfate formation in vivo. Our results indicate that adducted thumb-clubfoot syndrome is a disorder resulting from a defect specific to dermatan sulfate biosynthesis and emphasize roles for dermatan sulfate in human development and extracellular-matrix maintenance.
Authors:
Munis Dündar; Thomas Müller; Qi Zhang; Jing Pan; Beat Steinmann; Julia Vodopiutz; Robert Gruber; Tohru Sonoda; Birgit Krabichler; Gerd Utermann; Jacques U Baenziger; Lijuan Zhang; Andreas R Janecke
Publication Detail:
Type:  Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't    
Journal Detail:
Title:  American journal of human genetics     Volume:  85     ISSN:  1537-6605     ISO Abbreviation:  Am. J. Hum. Genet.     Publication Date:  2009 Dec 
Date Detail:
Created Date:  2009-12-16     Completed Date:  2010-01-14     Revised Date:  2010-12-03    
Medline Journal Info:
Nlm Unique ID:  0370475     Medline TA:  Am J Hum Genet     Country:  United States    
Other Details:
Languages:  eng     Pagination:  873-82     Citation Subset:  IM    
Affiliation:
Department of Medical Genetics, Erciyes University, Talas, 38039 Kayseri, Turkey.
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MeSH Terms
Descriptor/Qualifier:
Amino Acid Sequence
Clubfoot / genetics*
Craniofacial Abnormalities / genetics*
Disaccharides / metabolism
Epitopes
Hand Deformities / genetics*
Homozygote
Humans
Molecular Sequence Data
Mutation*
Mutation, Missense
Sequence Homology, Amino Acid
Sulfotransferases / genetics*,  physiology*
Syndrome
Thumb*
Grant Support
ID/Acronym/Agency:
R01 HD058474-19A1/HD/NICHD NIH HHS; R01DK41738/DK/NIDDK NIH HHS; R01GM069968/GM/NIGMS NIH HHS
Chemical
Reg. No./Substance:
0/Disaccharides; 0/Epitopes; EC 2.8.2.-/Sulfotransferases; EC 2.8.2.-/dermatan-4-sulfotransferase-1
Comments/Corrections

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