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Link to original content: http://pubmed.ncbi.nlm.nih.gov/22042873/
Garrod's fourth inborn error of metabolism solved by the identification of mutations causing pentosuria - PubMed Skip to main page content
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. 2011 Nov 8;108(45):18313-7.
doi: 10.1073/pnas.1115888108. Epub 2011 Oct 31.

Garrod's fourth inborn error of metabolism solved by the identification of mutations causing pentosuria

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Garrod's fourth inborn error of metabolism solved by the identification of mutations causing pentosuria

Sarah B Pierce et al. Proc Natl Acad Sci U S A. .

Abstract

Pentosuria is one of four conditions hypothesized by Archibald Garrod in 1908 to be inborn errors of metabolism. Mutations responsible for the other three conditions (albinism, alkaptonuria, and cystinuria) have been identified, but the mutations responsible for pentosuria remained unknown. Pentosuria, which affects almost exclusively individuals of Ashkenazi Jewish ancestry, is characterized by high levels of the pentose sugar L-xylulose in blood and urine and deficiency of the enzyme L-xylulose reductase. The condition is autosomal-recessive and completely clinically benign, but in the early and mid-20th century attracted attention because it was often confused with diabetes mellitus and inappropriately treated with insulin. Persons with pentosuria were identified from records of Margaret Lasker, who studied the condition in the 1930s to 1960s. In the DCXR gene encoding L-xylulose reductase, we identified two mutations, DCXR c.583ΔC and DCXR c.52(+1)G > A, each predicted to lead to loss of enzyme activity. Of nine unrelated living pentosuric subjects, six were homozygous for DCXR c.583ΔC, one was homozygous for DCXR c.52(+1)G > A, and two were compound heterozygous for the two mutant alleles. L-xylulose reductase was not detectable in protein lysates from subjects' cells and high levels of xylulose were detected in their sera, confirming the relationship between the DCXR genotypes and the pentosuric phenotype. The combined frequency of the two mutant DCXR alleles in 1,067 Ashkenazi Jewish controls was 0.0173, suggesting a pentosuria frequency of approximately one in 3,300 in this population. Haplotype analysis indicated that the DCXR c.52(+1)G > A mutation arose more recently than the DCXR c.583ΔC mutation.

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Conflict of interest statement

The authors declare no conflict of interest.

Figures

Fig. 1.
Fig. 1.
Pentosuria-associated mutations in the L-xylulose reductase gene DCXR. (A) Deletion of a single cytosine at chr17:79,994,115 (red arrow) corresponds to DCXR c.583ΔC. (B) Substitution at chr17:79,995,506 at the first base pair of intron 1 (red arrow) corresponds to DCXR c.52(+1)G > A. (C) Sketch of chromosome 17 indicating the position of DCXR in red. (D) Sketch of the DCXR gene, indicating the exon structure (blue bars), the translation start (green) and stop (red), and the positions of the two pentosuria-associated mutations.
Fig. 2.
Fig. 2.
Fifteen families from the series of Margaret Lasker, with pedigree structures as constructed by her at the dates indicated in parentheses. Asterisks (*) indicate persons tested for pentosuria in the 1950s; black symbols represent persons with pentosuria; white symbols represent persons without pentosuria; birth years are indicated under symbols. Arrows indicate participants in the present project. Recently available information indicates that families 75 and 83 are related, as shown. Family 80 has been updated to include participants born in the 1960s.
Fig. 3.
Fig. 3.
Mutant transcripts from the DCXR c.52(+1)G > A allele. Transcripts (n = 68) from this allele were cloned and sequenced. Exons 1 to 3 are diagrammed for the wild-type sequence. The site of the splice mutation in the genomic sequence is illustrated with a dashed line. Splice variant 1 (sv1, 41% of clones) extends exon 1 by 17 bp, skips exon 2, and splices into exon 3 at a site 120 bp after the canonical splice site. This variant creates an in-frame deletion of 68 aa. Splice variant 2 (sv2, 29% of clones) deletes 6 bp at the end of exon 1 and splices into exon 3 at the site 120 bp from the canonical splice. This variant introduces a frame-shift and premature stop (shown in red). Splice variant 3 (sv3, 12% of clones) deletes 6 bp at the 3′ end of exon 1 and splices canonically into exon 2 and then exon 3, creating an in-frame deletion of Lys17 and Gly18. The remaining 18% of clones represented multiple transcripts, each present once or twice, and all with aberrant splicing at the 3′ end of exon 1.
Fig. 4.
Fig. 4.
DCXR protein levels and plasma xylulose levels associated with DCXR genotypes. (A) Western blot of DCXR protein from lymphoblasts. Subjects with +/+ genotypes (lanes 1, 7, and 13) have normal DCXR sequence. Subjects with −/− genotypes are homozygous for c.52(+1)G > A (lane 2), homozygous for c.583ΔC (lanes 3–5), or compound heterozygous for c.52(+1)G > A/c.583ΔC (lane 6). Subjects with +/− genotypes are heterozygous for a wild-type allele and c.583ΔC (lanes 8–12). DCXR protein is undetectable in all individuals homozygous or compound heterozygous for mutant DCXR genotypes and reduced in heterozygous individuals relative to those with normal genotypes. HPRT was used as a loading control. (B) Xylulose levels detected by HILIC-APCI-MS in plasma of subjects of the DCXR genotypes defined above. Plasma of individuals of mutant (−/−) DCXR genotypes contains elevated xylulose, whereas xylulose levels are extremely low in plasma of individuals with heterozygous (+/−) or wild-type (+/+) genotypes.
Fig. 5.
Fig. 5.
Homozygosity mapping at DCXR on chromosome 17q25.3. The homozygous region surrounding DCXR for the subject homozygous for DCXR c.52(+1)G > A (green bar) is longer than those of subjects homozygous for DCXR c.583ΔC (blue bars), and much longer than those of Ashkenazi Jewish control subjects with wild-type DCXR sequences (mean length for 11 controls shown by the purple bar; details in Table S4). Position of the DCXR locus is indicated by the small black bar. Linkage disequilibrium values based on D′ from phased genotypes of individuals of European ancestry in the HapMap cohort (http://hapmap.ncbi.nlm.nih.gov) are shown in red. Relative lengths of the homozygous regions suggest that DCXR c.52(+1)G > A arose more recently than DCXR c.583ΔC and that both appeared after the European origin of the Ashkenazi Jewish population.

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