Literature DB >> 21872685

Systematic search for neutropenia should be part of the first screening in patients with poikiloderma.

Juliette Piard1, Muriel Holder-Espinasse, Bernard Aral, Nadège Gigot, Marlène Rio, Marc Tardieu, Eve Puzenat, Alice Goldenberg, Annick Toutain, Jerôme Franques, Kay MacDermot, Didier Bessis, Odile Boute, Patrick Callier, Lucie Gueneau, Frédéric Huet, Pierre Vabres, Benoît Catteau, Laurence Faivre, Christel Thauvin-Robinet.   

Abstract

Poikiloderma occurs in a number of hereditary syndromes, the best known of which is Rothmund-Thomson syndrome (RTS). Differential diagnoses include Dyskeratosis Congenita (DC) with high genetic heterogeneity and Clericuzio-type Poikiloderma with Neutropenia (CPN) due to mutations in the C16orf57 gene. Mutations in the RECQL4 gene are only observed in two thirds of RTS patients. In this study, 10 patients referred for syndromic poikiloderma and negative for RECQL4 sequencing analysis were investigated for C16orf57 mutations. Two C16orf57 heterozygous nonsense mutations (p.W81X and p.Y89X) were identified in a 5-year-old female child presenting with generalized poikiloderma, dental dysplasia, gingivitis, nail dystrophy, palmoplantar keratoderma and pachyonychia of the great toenails. Previously undetected and silent neutropenia was evidenced after C16orf57 molecular analysis. Neutropenia was absent in the C16orf57-negative patients. This report confirms that neutrophil count should be performed in all patients with poikiloderma to target the C16orf57 gene sequencing analysis, prior to RECQL4 analysis.
Copyright © 2011 Elsevier Masson SAS. All rights reserved.

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Year:  2011        PMID: 21872685     DOI: 10.1016/j.ejmg.2011.07.004

Source DB:  PubMed          Journal:  Eur J Med Genet        ISSN: 1769-7212            Impact factor:   2.708


  9 in total

1.  Incomplete splicing of neutrophil-specific genes affects neutrophil development in a zebrafish model of poikiloderma with neutropenia.

Authors:  Prakash Patil; Tamayo Uechi; Naoya Kenmochi
Journal:  RNA Biol       Date:  2015       Impact factor: 4.652

2.  Clinical utility gene card for: poikiloderma with neutropenia.

Authors:  Lidia Larizza; Gloria Negri; Elisa Adele Colombo; Ludovica Volpi; Yves Sznajer
Journal:  Eur J Hum Genet       Date:  2013-01-16       Impact factor: 4.246

3.  C16orf57, a gene mutated in poikiloderma with neutropenia, encodes a putative phosphodiesterase responsible for the U6 snRNA 3' end modification.

Authors:  Seweryn Mroczek; Joanna Krwawicz; Jan Kutner; Michal Lazniewski; Iwo Kuciński; Krzysztof Ginalski; Andrzej Dziembowski
Journal:  Genes Dev       Date:  2012-08-16       Impact factor: 11.361

4.  Insights into Mutation Effect in Three Poikiloderma with Neutropenia Patients by Transcript Analysis and Disease Evolution of Reported Patients with the Same Pathogenic Variants.

Authors:  Elisa A Colombo; Nursel H Elcioglu; Claudio Graziano; Pamela Farinelli; Elisabetta Di Fede; Iria Neri; Elena Facchini; Mariangela Greco; Cristina Gervasini; Lidia Larizza
Journal:  J Clin Immunol       Date:  2018-05-16       Impact factor: 8.317

5.  Novel C16orf57 mutations in patients with Poikiloderma with Neutropenia: bioinformatic analysis of the protein and predicted effects of all reported mutations.

Authors:  Elisa A Colombo; J Fernando Bazan; Gloria Negri; Cristina Gervasini; Nursel H Elcioglu; Deniz Yucelten; Ilknur Altunay; Umram Cetincelik; Anna Teti; Andrea Del Fattore; Matteo Luciani; Spencer K Sullivan; Albert C Yan; Ludovica Volpi; Lidia Larizza
Journal:  Orphanet J Rare Dis       Date:  2012-01-23       Impact factor: 4.123

6.  Expanding the clinical spectrum of hereditary fibrosing poikiloderma with tendon contractures, myopathy and pulmonary fibrosis due to FAM111B mutations.

Authors:  Sandra Mercier; Sébastien Küry; Emmanuelle Salort-Campana; Armelle Magot; Uchenna Agbim; Thomas Besnard; Nathalie Bodak; Chantal Bou-Hanna; Flora Bréhéret; Perrine Brunelle; Florence Caillon; Brigitte Chabrol; Valérie Cormier-Daire; Albert David; Bruno Eymard; Laurence Faivre; Dominique Figarella-Branger; Emmanuelle Fleurence; Mythily Ganapathi; Romain Gherardi; Alice Goldenberg; Antoine Hamel; Jeanine Igual; Alan D Irvine; Dominique Israël-Biet; Caroline Kannengiesser; Christian Laboisse; Cédric Le Caignec; Jean-Yves Mahé; Stéphanie Mallet; Stuart MacGowan; Maeve A McAleer; Irwin McLean; Cécile Méni; Arnold Munnich; Jean-Marie Mussini; Peter L Nagy; Jeffrey Odel; Grainne M O'Regan; Yann Péréon; Julie Perrier; Juliette Piard; Eve Puzenat; Jacinda B Sampson; Frances Smith; Nadem Soufir; Kurenai Tanji; Christel Thauvin; Christina Ulane; Rosemarie M Watson; Nonhlanhla P Khumalo; Bongani M Mayosi; Sébastien Barbarot; Stéphane Bézieau
Journal:  Orphanet J Rare Dis       Date:  2015-10-15       Impact factor: 4.123

7.  Mutations in FAM111B cause hereditary fibrosing poikiloderma with tendon contracture, myopathy, and pulmonary fibrosis.

Authors:  Sandra Mercier; Sébastien Küry; Gasnat Shaboodien; Darren T Houniet; Nonhlanhla P Khumalo; Chantal Bou-Hanna; Nathalie Bodak; Valérie Cormier-Daire; Albert David; Laurence Faivre; Dominique Figarella-Branger; Romain K Gherardi; Elise Glen; Antoine Hamel; Christian Laboisse; Cédric Le Caignec; Pierre Lindenbaum; Armelle Magot; Arnold Munnich; Jean-Marie Mussini; Komala Pillay; Thahira Rahman; Richard Redon; Emmanuelle Salort-Campana; Mauro Santibanez-Koref; Christel Thauvin; Sébastien Barbarot; Bernard Keavney; Stéphane Bézieau; Bongani M Mayosi
Journal:  Am J Hum Genet       Date:  2013-11-21       Impact factor: 11.025

8.  Poikiloderma With Neutropenia and Mastocytosis: A Case Report and a Review of Dermatological Signs.

Authors:  Vincenzo Piccolo; Teresa Russo; Daniela Di Pinto; Elvira Pota; Martina Di Martino; Giulio Piluso; Andrea Ronchi; Giuseppe Argenziano; Eugenia Veronica Di Brizzi; Claudia Santoro
Journal:  Front Med (Lausanne)       Date:  2021-06-10

9.  A zebrafish model of Poikiloderma with Neutropenia recapitulates the human syndrome hallmarks and traces back neutropenia to the myeloid progenitor.

Authors:  Elisa A Colombo; Silvia Carra; Laura Fontana; Erica Bresciani; Franco Cotelli; Lidia Larizza
Journal:  Sci Rep       Date:  2015-11-02       Impact factor: 4.379

  9 in total

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