Literature DB >> 32903913

Renpenning Syndrome in a Turkish Patient: de novo Variant c.607C>T in PACS1 and Hypogammaglobulinemia Phenotype.

Fatma Kurt Colak1, Nilnur Eyerci2, Caner Aytekin3, Ayse S Eksioglu4.   

Abstract

Renpenning syndrome is an X-linked intellectual disability syndrome caused by mutations in the human polyglutamine binding protein 1 (PQBP1) gene characterized by intellectual disability (ID), microcephaly, and dysmorphic facial features. We report a Turkish child with a novel pathogenic variant in PQBP1 and a likely pathogenic variant in the PACS1 gene presenting with growth restriction, microcephaly, ID, micropenis, bilateral iris coloboma, and hypogammaglobulinemia. Cytogenetic investigations, including a high-resolution-banded karyotype, were normal. Clinical exome sequencing was performed. We found the novel PQBP1 variant, c.640C>T; p.(Arg214Trp), and the known PACS1 variant, c.607C>T; p.(Arg203Trp), in the proband. The patient's hypogammaglobulinemia did not respond to treatment. This condition was detected for the first time in a patient with Renpenning syndrome.
Copyright © 2020 by S. Karger AG, Basel.

Entities:  

Keywords:  Novel variant; PACS1; PQBP1; Renpenning syndrome; c.640C>T

Year:  2020        PMID: 32903913      PMCID: PMC7445573          DOI: 10.1159/000507562

Source DB:  PubMed          Journal:  Mol Syndromol        ISSN: 1661-8769


  10 in total

1.  Clinical delineation of the PACS1-related syndrome--Report on 19 patients.

Authors:  Janneke H M Schuurs-Hoeijmakers; Megan L Landsverk; Nicola Foulds; Mary K Kukolich; Ralitza H Gavrilova; Stephanie Greville-Heygate; Andrea Hanson-Kahn; Jonathan A Bernstein; Jennifer Glass; David Chitayat; Thomas A Burrow; Ammar Husami; Kathleen Collins; Katie Wusik; Nathalie van der Aa; Frank Kooy; Kate Tatton Brown; Dorothea Gadzicki; Usha Kini; Sara Alvarez; Alberto Fernández-Jaén; Frank McGehee; Katherine Selby; Maja Tarailo-Graovac; Margot Van Allen; Clara D M van Karnebeek; Dimitri J Stavropoulos; Christian R Marshall; Daniele Merico; Anne Gregor; Christiane Zweier; Robert J Hopkin; Yoyo Wing-Yiu Chu; Brian Hon-Yin Chung; Bert B A de Vries; Koenraad Devriendt; Matthew E Hurles; Han G Brunner
Journal:  Am J Med Genet A       Date:  2016-02-03       Impact factor: 2.802

2.  Expanding the phenotype of a recurrent de novo variant in PACS1 causing intellectual disability.

Authors:  D Gadzicki; D Döcker; M Schubach; M Menzel; B Schmorl; F Stellmer; S Biskup; D Bartholdi
Journal:  Clin Genet       Date:  2014-12-18       Impact factor: 4.438

3.  PQBP-1/Npw38, a nuclear protein binding to the polyglutamine tract, interacts with U5-15kD/dim1p via the carboxyl-terminal domain.

Authors:  M Waragai; E Junn; M Kajikawa; S Takeuchi; I Kanazawa; M Shibata; M M Mouradian; H Okazawa
Journal:  Biochem Biophys Res Commun       Date:  2000-07-05       Impact factor: 3.575

Review 4.  PQBP1, an intrinsically disordered/denatured protein at the crossroad of intellectual disability and neurodegenerative diseases.

Authors:  Hitoshi Okazawa
Journal:  Neurochem Int       Date:  2017-06-13       Impact factor: 3.921

5.  Renpenning syndrome comes into focus.

Authors:  Roger E Stevenson; C W Bennett; F Abidi; T Kleefstra; M Porteous; R J Simensen; H A Lubs; B C J Hamel; C E Schwartz
Journal:  Am J Med Genet A       Date:  2005-05-01       Impact factor: 2.802

6.  The Renpenning syndrome spectrum: new clinical insights supported by 13 new PQBP1-mutated males.

Authors:  D Germanaud; M Rossi; G Bussy; D Gérard; L Hertz-Pannier; P Blanchet; H Dollfus; F Giuliano; V Bennouna-Greene; P Sarda; S Sigaudy; A Curie; M C Vincent; R Touraine; V des Portes
Journal:  Clin Genet       Date:  2010-10-18       Impact factor: 4.438

7.  Recurrent de novo mutations in PACS1 cause defective cranial-neural-crest migration and define a recognizable intellectual-disability syndrome.

Authors:  Janneke H M Schuurs-Hoeijmakers; Edwin C Oh; Lisenka E L M Vissers; Mariëlle E M Swinkels; Christian Gilissen; Michèl A Willemsen; Maureen Holvoet; Marloes Steehouwer; Joris A Veltman; Bert B A de Vries; Hans van Bokhoven; Arjan P M de Brouwer; Nicholas Katsanis; Koenraad Devriendt; Han G Brunner
Journal:  Am J Hum Genet       Date:  2012-11-15       Impact factor: 11.025

8.  ACMG recommendations for standards for interpretation and reporting of sequence variations: Revisions 2007.

Authors:  C Sue Richards; Sherri Bale; Daniel B Bellissimo; Soma Das; Wayne W Grody; Madhuri R Hegde; Elaine Lyon; Brian E Ward
Journal:  Genet Med       Date:  2008-04       Impact factor: 8.822

9.  Nematode homologue of PQBP1, a mental retardation causative gene, is involved in lipid metabolism.

Authors:  Keiko Takahashi; Sawako Yoshina; Maekawa Masashi; Wakana Ito; Takao Inoue; Hiroki Shiwaku; Hiroyuki Arai; Shohei Mitani; Hitoshi Okazawa
Journal:  PLoS One       Date:  2009-01-01       Impact factor: 3.240

10.  Cross-Tissue Regulatory Gene Networks in Coronary Artery Disease.

Authors:  Husain A Talukdar; Hassan Foroughi Asl; Rajeev K Jain; Raili Ermel; Arno Ruusalepp; Oscar Franzén; Brian A Kidd; Ben Readhead; Chiara Giannarelli; Jason C Kovacic; Torbjörn Ivert; Joel T Dudley; Mete Civelek; Aldons J Lusis; Eric E Schadt; Josefin Skogsberg; Tom Michoel; Johan L M Björkegren
Journal:  Cell Syst       Date:  2016-03-03       Impact factor: 10.304

  10 in total
  1 in total

1.  Schuurs-Hoeijmakers Syndrome (PACS1 Neurodevelopmental Disorder): Seven Novel Patients and a Review.

Authors:  Jair Tenorio-Castaño; Beatriz Morte; Julián Nevado; Víctor Martinez-Glez; Fernando Santos-Simarro; Sixto García-Miñaúr; María Palomares-Bralo; Marta Pacio-Míguez; Beatriz Gómez; Pedro Arias; Alba Alcochea; Juan Carrión; Patricia Arias; Berta Almoguera; Fermina López-Grondona; Isabel Lorda-Sanchez; Enrique Galán-Gómez; Irene Valenzuela; María Pilar Méndez Perez; Ivón Cuscó; Francisco Barros; Juan Pié; Sergio Ramos; Feliciano J Ramos; Alma Kuechler; Eduardo Tizzano; Carmen Ayuso; Frank J Kaiser; Luis A Pérez-Jurado; Ángel Carracedo; Pablo Lapunzina
Journal:  Genes (Basel)       Date:  2021-05-13       Impact factor: 4.096

  1 in total

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