| Literature DB >> 32903913 |
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.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