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Goltz–Gorlin (focal dermal hypoplasia) and the microphthalmia with linear skin defects (MLS) syndrome: no evidence of genetic overlap

Identifieur interne : 000047 ( Pmc/Corpus ); précédent : 000046; suivant : 000048

Goltz–Gorlin (focal dermal hypoplasia) and the microphthalmia with linear skin defects (MLS) syndrome: no evidence of genetic overlap

Auteurs : May-Britt Harmsen ; Silvia Azzarello-Burri ; M Mar García González ; Gabriele Gillessen-Kaesbach ; Peter Meinecke ; Dietmar Müller ; Anita Rauch ; Eva Rossier ; Eva Seemanova ; Christiane Spaich ; Bernhard Steiner ; Dagmar Wieczorek ; Martin Zenker ; Kerstin Kutsche

Source :

RBID : PMC:2986635

Abstract

Focal dermal hypoplasia (FDH) is an X-linked developmental disorder with male lethality characterized by patchy dermal hypoplasia, skeletal and dental malformations, and microphthalmia or anophthalmia. Recently, heterozygous loss-of-function mutations in the PORCN gene have been described to cause FDH. FDH shows some clinical overlap with the microphthalmia with linear skin defects (MLS) syndrome, another X-linked male lethal condition, associated with mutations of HCCS in the majority of cases. We performed DNA sequencing of PORCN in 13 female patients with the clinical diagnosis of FDH as well as four female patients with MLS syndrome and no mutation in HCCS. We identified PORCN mutations in all female patients with FDH. Eleven patients seem to have constitutional PORCN alterations in the heterozygous state and two individuals are mosaic for the heterozygous sequence change in PORCN. No PORCN mutation was identified in the MLS-affected patients, providing further evidence that FDH and MLS do not overlap genetically. X chromosome inactivation (XCI) analysis revealed a random or slightly skewed XCI pattern in leukocytes of individuals with intragenic PORCN mutation suggesting that defective PORCN does not lead to selective growth disadvantage, at least in leukocytes. We conclude that the PORCN mutation detection rate is high in individuals with a clear-cut FDH phenotype and somatic mosaicism can be present in a significant proportion of patients with mild or classic FDH.


Url:
DOI: 10.1038/ejhg.2009.40
PubMed: 19277062
PubMed Central: 2986635

Links to Exploration step

PMC:2986635

Le document en format XML

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<name sortKey="Azzarello Burri, Silvia" sort="Azzarello Burri, Silvia" uniqKey="Azzarello Burri S" first="Silvia" last="Azzarello-Burri">Silvia Azzarello-Burri</name>
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, Figueres,
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<name sortKey="Gillessen Kaesbach, Gabriele" sort="Gillessen Kaesbach, Gabriele" uniqKey="Gillessen Kaesbach G" first="Gabriele" last="Gillessen-Kaesbach">Gabriele Gillessen-Kaesbach</name>
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<name sortKey="Rauch, Anita" sort="Rauch, Anita" uniqKey="Rauch A" first="Anita" last="Rauch">Anita Rauch</name>
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<name sortKey="Spaich, Christiane" sort="Spaich, Christiane" uniqKey="Spaich C" first="Christiane" last="Spaich">Christiane Spaich</name>
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<name sortKey="Wieczorek, Dagmar" sort="Wieczorek, Dagmar" uniqKey="Wieczorek D" first="Dagmar" last="Wieczorek">Dagmar Wieczorek</name>
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<p>Focal dermal hypoplasia (FDH) is an X-linked developmental disorder with male lethality characterized by patchy dermal hypoplasia, skeletal and dental malformations, and microphthalmia or anophthalmia. Recently, heterozygous loss-of-function mutations in the
<italic>PORCN</italic>
gene have been described to cause FDH. FDH shows some clinical overlap with the microphthalmia with linear skin defects (MLS) syndrome, another X-linked male lethal condition, associated with mutations of
<italic>HCCS</italic>
in the majority of cases. We performed DNA sequencing of
<italic>PORCN</italic>
in 13 female patients with the clinical diagnosis of FDH as well as four female patients with MLS syndrome and no mutation in
<italic>HCCS</italic>
. We identified
<italic>PORCN</italic>
mutations in all female patients with FDH. Eleven patients seem to have constitutional
<italic>PORCN</italic>
alterations in the heterozygous state and two individuals are mosaic for the heterozygous sequence change in
<italic>PORCN</italic>
. No
<italic>PORCN</italic>
mutation was identified in the MLS-affected patients, providing further evidence that FDH and MLS do not overlap genetically. X chromosome inactivation (XCI) analysis revealed a random or slightly skewed XCI pattern in leukocytes of individuals with intragenic
<italic>PORCN</italic>
mutation suggesting that defective PORCN does not lead to selective growth disadvantage, at least in leukocytes. We conclude that the
<italic>PORCN</italic>
mutation detection rate is high in individuals with a clear-cut FDH phenotype and somatic mosaicism can be present in a significant proportion of patients with mild or classic FDH.</p>
</div>
</front>
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<italic>PORCN</italic>
mutations in patients with FDH</alt-title>
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<name>
<surname>Harmsen</surname>
<given-names>May-Britt</given-names>
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<xref ref-type="aff" rid="aff1">1</xref>
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<contrib contrib-type="author">
<name>
<surname>Azzarello-Burri</surname>
<given-names>Silvia</given-names>
</name>
<xref ref-type="aff" rid="aff2">2</xref>
</contrib>
<contrib contrib-type="author">
<name>
<surname>García González</surname>
<given-names>M Mar</given-names>
</name>
<xref ref-type="aff" rid="aff3">3</xref>
</contrib>
<contrib contrib-type="author">
<name>
<surname>Gillessen-Kaesbach</surname>
<given-names>Gabriele</given-names>
</name>
<xref ref-type="aff" rid="aff4">4</xref>
</contrib>
<contrib contrib-type="author">
<name>
<surname>Meinecke</surname>
<given-names>Peter</given-names>
</name>
<xref ref-type="aff" rid="aff5">5</xref>
</contrib>
<contrib contrib-type="author">
<name>
<surname>Müller</surname>
<given-names>Dietmar</given-names>
</name>
<xref ref-type="aff" rid="aff6">6</xref>
</contrib>
<contrib contrib-type="author">
<name>
<surname>Rauch</surname>
<given-names>Anita</given-names>
</name>
<xref ref-type="aff" rid="aff7">7</xref>
</contrib>
<contrib contrib-type="author">
<name>
<surname>Rossier</surname>
<given-names>Eva</given-names>
</name>
<xref ref-type="aff" rid="aff8">8</xref>
</contrib>
<contrib contrib-type="author">
<name>
<surname>Seemanova</surname>
<given-names>Eva</given-names>
</name>
<xref ref-type="aff" rid="aff9">9</xref>
</contrib>
<contrib contrib-type="author">
<name>
<surname>Spaich</surname>
<given-names>Christiane</given-names>
</name>
<xref ref-type="aff" rid="aff10">10</xref>
</contrib>
<contrib contrib-type="author">
<name>
<surname>Steiner</surname>
<given-names>Bernhard</given-names>
</name>
<xref ref-type="aff" rid="aff2">2</xref>
</contrib>
<contrib contrib-type="author">
<name>
<surname>Wieczorek</surname>
<given-names>Dagmar</given-names>
</name>
<xref ref-type="aff" rid="aff11">11</xref>
</contrib>
<contrib contrib-type="author">
<name>
<surname>Zenker</surname>
<given-names>Martin</given-names>
</name>
<xref ref-type="aff" rid="aff7">7</xref>
</contrib>
<contrib contrib-type="author">
<name>
<surname>Kutsche</surname>
<given-names>Kerstin</given-names>
</name>
<xref ref-type="aff" rid="aff1">1</xref>
<xref ref-type="corresp" rid="caf1">*</xref>
</contrib>
<aff id="aff1">
<label>1</label>
<institution>Institut für Humangenetik, Universitätsklinikum Hamburg-Eppendorf</institution>
, Hamburg,
<country>Germany</country>
</aff>
<aff id="aff2">
<label>2</label>
<institution>Institut für Medizinische Genetik, Universität Zürich</institution>
, Schwerzenbach,
<country>Switzerland</country>
</aff>
<aff id="aff3">
<label>3</label>
<institution>Servei de Pediatria, Hospital de Figueres</institution>
, Figueres,
<country>Spain</country>
</aff>
<aff id="aff4">
<label>4</label>
<institution>Institut für Humangenetik, Universität zu Lübeck</institution>
, Lübeck,
<country>Germany</country>
</aff>
<aff id="aff5">
<label>5</label>
<institution>Medizinische Genetik, Altonaer Kinderkrankenhaus</institution>
, Hamburg,
<country>Germany</country>
</aff>
<aff id="aff6">
<label>6</label>
<institution>Institut für Medizinische Genetik, Klinikum Chemnitz</institution>
, Chemnitz,
<country>Germany</country>
</aff>
<aff id="aff7">
<label>7</label>
<institution>Humangenetisches Institut, Universitätsklinikum Erlangen</institution>
, Erlangen,
<country>Germany</country>
</aff>
<aff id="aff8">
<label>8</label>
<institution>Medizinische Genetik, Universitätsklinikum Tübingen</institution>
, Tübingen,
<country>Germany</country>
</aff>
<aff id="aff9">
<label>9</label>
<institution>Department of Clinical Genetics, Institute of Biology and Medical Genetics, Charles University</institution>
, Prague,
<country>Czech Republic</country>
</aff>
<aff id="aff10">
<label>10</label>
<institution>Institut für Klinische Genetik, Klinikum Stuttgart</institution>
, Stuttgart,
<country>Germany</country>
</aff>
<aff id="aff11">
<label>11</label>
<institution>Institut für Humangenetik, Universitätsklinikum Essen</institution>
, Essen,
<country>Germany</country>
</aff>
</contrib-group>
<author-notes>
<corresp id="caf1">
<label>*</label>
<institution>Institut für Humangenetik, Universitätsklinikum Hamburg-Eppendorf</institution>
, Campus Forschung, Martinistrasse 52, 20246 Hamburg,
<country>Germany</country>
. Tel: +49 40 7410 54597; Fax: +49 40 7410 55138; E-mail:
<email>kkutsche@uke.de</email>
</corresp>
</author-notes>
<pub-date pub-type="ppub">
<month>10</month>
<year>2009</year>
</pub-date>
<pub-date pub-type="epub">
<day>11</day>
<month>03</month>
<year>2009</year>
</pub-date>
<volume>17</volume>
<issue>10</issue>
<fpage>1207</fpage>
<lpage>1215</lpage>
<history>
<date date-type="received">
<day>29</day>
<month>07</month>
<year>2008</year>
</date>
<date date-type="rev-recd">
<day>09</day>
<month>02</month>
<year>2009</year>
</date>
<date date-type="accepted">
<day>11</day>
<month>02</month>
<year>2009</year>
</date>
</history>
<permissions>
<copyright-statement>Copyright © 2009 Macmillan Publishers Limited</copyright-statement>
<copyright-year>2009</copyright-year>
<copyright-holder>Macmillan Publishers Limited</copyright-holder>
</permissions>
<abstract>
<p>Focal dermal hypoplasia (FDH) is an X-linked developmental disorder with male lethality characterized by patchy dermal hypoplasia, skeletal and dental malformations, and microphthalmia or anophthalmia. Recently, heterozygous loss-of-function mutations in the
<italic>PORCN</italic>
gene have been described to cause FDH. FDH shows some clinical overlap with the microphthalmia with linear skin defects (MLS) syndrome, another X-linked male lethal condition, associated with mutations of
<italic>HCCS</italic>
in the majority of cases. We performed DNA sequencing of
<italic>PORCN</italic>
in 13 female patients with the clinical diagnosis of FDH as well as four female patients with MLS syndrome and no mutation in
<italic>HCCS</italic>
. We identified
<italic>PORCN</italic>
mutations in all female patients with FDH. Eleven patients seem to have constitutional
<italic>PORCN</italic>
alterations in the heterozygous state and two individuals are mosaic for the heterozygous sequence change in
<italic>PORCN</italic>
. No
<italic>PORCN</italic>
mutation was identified in the MLS-affected patients, providing further evidence that FDH and MLS do not overlap genetically. X chromosome inactivation (XCI) analysis revealed a random or slightly skewed XCI pattern in leukocytes of individuals with intragenic
<italic>PORCN</italic>
mutation suggesting that defective PORCN does not lead to selective growth disadvantage, at least in leukocytes. We conclude that the
<italic>PORCN</italic>
mutation detection rate is high in individuals with a clear-cut FDH phenotype and somatic mosaicism can be present in a significant proportion of patients with mild or classic FDH.</p>
</abstract>
<kwd-group>
<kwd>FDH</kwd>
<kwd>male-lethal</kwd>
<kwd>MIDAS</kwd>
<kwd>mosaicism</kwd>
<kwd>X-chromosomal dominant</kwd>
<kwd>X-inactivation</kwd>
</kwd-group>
</article-meta>
</front>
</pmc>
</record>

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