Abstract
Thiamine-responsive megaloblastic anaemia (TRMA) syndrome with diabetes and deafness was found in two patients from a Tunisian kindred. The proband was homozygous for a novel mutation, 287delG, in the high-affinity thiamine transporter gene, SLC19A2. We demonstrated that fibroblasts from this patient exhibited defective thiamine transport. These data confirm that the SLC19A2 gene is the high-affinity thiamine carrier and that this novel mutation is responsible for TRMA syndrome.
Publication types
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Case Reports
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Research Support, Non-U.S. Gov't
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Research Support, U.S. Gov't, P.H.S.
MeSH terms
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Anemia, Megaloblastic / drug therapy
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Anemia, Megaloblastic / genetics*
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Anemia, Megaloblastic / pathology
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Bone Marrow Cells / pathology
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Cardiomyopathy, Dilated / genetics
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Cardiomyopathy, Dilated / pathology
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Carrier Proteins / genetics*
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Child, Preschool
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Deafness / genetics*
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Deafness / pathology
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Diabetes Mellitus, Type 1 / genetics*
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Diabetes Mellitus, Type 1 / pathology
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Female
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Haplotypes
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Humans
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Infant
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Male
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Membrane Transport Proteins*
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Pedigree
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Point Mutation*
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Syndrome
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Thiamine / therapeutic use
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Tunisia
Substances
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Carrier Proteins
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Membrane Transport Proteins
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SLC19A2 protein, human
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Thiamine