Literature DB >> 28817236

A new CUL4B variant associated with a mild phenotype and an exceptional pattern of leukoencephalopathy.

Susann Weissbach1, Marie-Christine Reinert1, Janine Altmüller2, Ralph Krätzner1, Holger Thiele2, Thorsten Rosenbaum3, Peter Nürnberg2, Jutta Gärtner1.   

Abstract

Cabezas type of X-linked syndromic intellectual disability (MRXSC; MIM300354) is a rare X-linked recessive intellectual disability characterized primarily by intellectual disability, short stature, hypogonadism, and gait abnormalities. It is caused by a wide spectrum of hemizygous variants in CUL4B. In a 10-year-old boy with an exceptional leukoencephalopathy pattern, we identified a new missense variant p.Leu329Gln in CUL4B using "Mendeliome" sequencing. However, his phenotype does not include the severe characteristics currently known for MRXSC. We discuss the divergent phenotype and propose a potential connection between the different CUL4B variants and corresponding phenotypes in the context of the current literature as well as 3D homology modeling.
© 2017 Wiley Periodicals, Inc.

Entities:  

Keywords:  CUL4B; Cabezas type; cullin 4B; gait abnormalities; leukoencephalopathy; white matter lesion

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Substances:

Year:  2017        PMID: 28817236     DOI: 10.1002/ajmg.a.38390

Source DB:  PubMed          Journal:  Am J Med Genet A        ISSN: 1552-4825            Impact factor:   2.802


  3 in total

1.  Late diagnosis of a truncating WISP3 mutation entails a severe phenotype of progressive pseudorheumatoid dysplasia.

Authors:  Salem Alawbathani; Amit Kawalia; Mert Karakaya; Janine Altmüller; Peter Nürnberg; Sebahattin Cirak
Journal:  Cold Spring Harb Mol Case Stud       Date:  2018-02-01

2.  A novel CUL4B splice site variant in a young male exhibiting less pronounced features.

Authors:  Yuji Nakamura; Yusuke Okuno; Hideki Muramatsu; Tomoko Kawai; Kazuhito Satou; Daisuke Ieda; Ikumi Hori; Kei Ohashi; Yutaka Negishi; Ayako Hattori; Yoshiyuki Takahashi; Seiji Kojima; Shinji Saitoh
Journal:  Hum Genome Var       Date:  2019-09-04

Review 3.  The Ubiquitin System: a Regulatory Hub for Intellectual Disability and Autism Spectrum Disorder.

Authors:  Stephen A Wood; Michael Piper; Maria A Kasherman; Susitha Premarathne; Thomas H J Burne
Journal:  Mol Neurobiol       Date:  2020-01-23       Impact factor: 5.590

  3 in total

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