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SALL1 mutation analysis in Townes-Brocks syndrome: twelve novel mutations and expansion of the phenotype

✍ Scribed by Elke M. Botzenhart; Andrew Green; Helena Ilyina; Rainer König; R. Brian Lowry; Ivan F. M. Lo; Mordechai Shohat; Leah Burke; Julie McGaughran; Ronit Chafai; Geneviève Pierquin; Ron C Michaelis; Margo L. Whiteford; Kalle O. J. Simola; Bernd Rösler; Jürgen Kohlhase


Publisher
John Wiley and Sons
Year
2005
Tongue
English
Weight
148 KB
Volume
26
Category
Article
ISSN
1059-7794

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✦ Synopsis


Townes-Brocks syndrome is an autosomal dominantly inherited disorder, which comprises multiple birth defects including renal, ear, anal, and limb malformations. TBS has been shown to result from mutations in SALL1, a human gene related to the developmental regulator SAL of Drosophila melanogaster. The SALL1 gene product is a zinc finger protein thought to act as a transcription factor. It contains four highly conserved, evenly distributed C2H2 double zinc finger domains. A single C2H2 motif is attached to the second domain, and at the amino terminus SALL1 contains a C2HC motif. Most mutations causing TBS are clustered in the N-terminal third of the SALL1 coding region and result in the production of truncated proteins containing only one or none of the C2H2 domains and the N-terminal transcriptional repressor domain of SALL1. Twenty-three SALL1 mutations were reported prior to this work, 22 of which are located in exon 2, 5' of the second double zinc finger-encoding region. Here we present 12 novel mutations in SALL1 associated with Townes-Brocks syndrome in 13 unrelated families. These include three nonsense mutations, three short insertions and six short deletions. Thus the number of SALL1 mutations increases to 35. Rare phenotypical features among mutation positive patients include hypothyroidism, vaginal aplasia with bifid uterus, cryptorchidism, bifid scrotum without hypospadia scrotalis, unilateral chorioretinal coloboma with loss of vision, dorsal hypoplasia of the corpus callosum, and umbilical hernia.


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Townes-Brocks syndrome: twenty novel SAL
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Townes-Brocks syndrome (TBS) is an autosomal dominant malformation syndrome characterized by renal, anal, ear, and thumb anomalies caused by SALL1 mutations. To date, 36 SALL1 mutations have been described in TBS patients. All but three of those, namely p.R276X, p.S372X, and c.1404dupG, have been fo

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Here we report on a 3-year-old boy with full-blown TBS and two rare features, Duane anomaly (DA) and somatic mosaicism for the causative SALL1 mutation. This boy is the third child of non-consanguineous parents and he was born at 39 þ6 weeks gestation as the second child of a dizygotic twin pregnan