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LGMD1D myopathy with cytoplasmic and nuclear inclusions in a Saudi family due to DNAJB6 mutation

Autosomal dominant LGMD1D has been described in multiple families in Asia, Europe, and USA. However, to the best of our knowledge, no cases of LGMD1D have been reported among native Bedouin Saudi families. Fifty Saudi families with LGMD were analyzed and the causative underlying genes were studied u...

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Autores principales: BOHLEGA, SAEED A., ALFAWAZ, SARAH, ABOU-AL-SHAAR, HUSSAM, AL-HINDI, HINDI N., MURAD, HATEM N., BOHLEGA, MOHAMED S., MEYER, BRIAN F., MONIES, DOROTA
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Pacini Editore srl 2018
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6390114/
https://www.ncbi.nlm.nih.gov/pubmed/30838352
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author BOHLEGA, SAEED A.
ALFAWAZ, SARAH
ABOU-AL-SHAAR, HUSSAM
AL-HINDI, HINDI N.
MURAD, HATEM N.
BOHLEGA, MOHAMED S.
MEYER, BRIAN F.
MONIES, DOROTA
author_facet BOHLEGA, SAEED A.
ALFAWAZ, SARAH
ABOU-AL-SHAAR, HUSSAM
AL-HINDI, HINDI N.
MURAD, HATEM N.
BOHLEGA, MOHAMED S.
MEYER, BRIAN F.
MONIES, DOROTA
author_sort BOHLEGA, SAEED A.
collection PubMed
description Autosomal dominant LGMD1D has been described in multiple families in Asia, Europe, and USA. However, to the best of our knowledge, no cases of LGMD1D have been reported among native Bedouin Saudi families. Fifty Saudi families with LGMD were analyzed and the causative underlying genes were studied utilizing genome wide linkage, homozygosity mapping, and neurological gene panel. We identified one family of a Bedouin origin with LGMD1D. Two patients had progressive proximal and distal weakness, dysphagia, and respiratory symptoms. Creatinine kinase was normal. Muscle biopsy showed marked variation in myofibers size with scattered angular atrophic fiber, necrotic fibers, and myophagocytosis, with red-rimmed vacuoles depicting a sarcoplasmic body. Heterozygous c.C287T (p.P96L) variant in exon 5 of DNAJB6 (NM_005494) gene was found. This change is localized within glycine and phenylalanine rich domain and alter an amino acid residue. Our findings will expand on the existing genotypic and phenotypic spectrum of this disorder and aid in elucidating hidden mechanisms implicated in LGMD1D.
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spelling pubmed-63901142019-03-05 LGMD1D myopathy with cytoplasmic and nuclear inclusions in a Saudi family due to DNAJB6 mutation BOHLEGA, SAEED A. ALFAWAZ, SARAH ABOU-AL-SHAAR, HUSSAM AL-HINDI, HINDI N. MURAD, HATEM N. BOHLEGA, MOHAMED S. MEYER, BRIAN F. MONIES, DOROTA Acta Myol Original Article Autosomal dominant LGMD1D has been described in multiple families in Asia, Europe, and USA. However, to the best of our knowledge, no cases of LGMD1D have been reported among native Bedouin Saudi families. Fifty Saudi families with LGMD were analyzed and the causative underlying genes were studied utilizing genome wide linkage, homozygosity mapping, and neurological gene panel. We identified one family of a Bedouin origin with LGMD1D. Two patients had progressive proximal and distal weakness, dysphagia, and respiratory symptoms. Creatinine kinase was normal. Muscle biopsy showed marked variation in myofibers size with scattered angular atrophic fiber, necrotic fibers, and myophagocytosis, with red-rimmed vacuoles depicting a sarcoplasmic body. Heterozygous c.C287T (p.P96L) variant in exon 5 of DNAJB6 (NM_005494) gene was found. This change is localized within glycine and phenylalanine rich domain and alter an amino acid residue. Our findings will expand on the existing genotypic and phenotypic spectrum of this disorder and aid in elucidating hidden mechanisms implicated in LGMD1D. Pacini Editore srl 2018-09-01 /pmc/articles/PMC6390114/ /pubmed/30838352 Text en ©2018 Gaetano Conte Academy, Naples, Italy http://creativecommons.org/licenses/by-nc-nd/4.0/ This is an Open Access article distributed under the terms of the Creative Commons Attribution NonCommercial-NoDerivatives 4.0 International (CC BY-NC-ND 4.0), which permits for noncommercial use, distribution, and reproduction in any digital medium, provided the original work is properly cited and is not altered in any way. For details, please refer to https://creativecommons.org/licenses/by-nc-nd/4.0/
spellingShingle Original Article
BOHLEGA, SAEED A.
ALFAWAZ, SARAH
ABOU-AL-SHAAR, HUSSAM
AL-HINDI, HINDI N.
MURAD, HATEM N.
BOHLEGA, MOHAMED S.
MEYER, BRIAN F.
MONIES, DOROTA
LGMD1D myopathy with cytoplasmic and nuclear inclusions in a Saudi family due to DNAJB6 mutation
title LGMD1D myopathy with cytoplasmic and nuclear inclusions in a Saudi family due to DNAJB6 mutation
title_full LGMD1D myopathy with cytoplasmic and nuclear inclusions in a Saudi family due to DNAJB6 mutation
title_fullStr LGMD1D myopathy with cytoplasmic and nuclear inclusions in a Saudi family due to DNAJB6 mutation
title_full_unstemmed LGMD1D myopathy with cytoplasmic and nuclear inclusions in a Saudi family due to DNAJB6 mutation
title_short LGMD1D myopathy with cytoplasmic and nuclear inclusions in a Saudi family due to DNAJB6 mutation
title_sort lgmd1d myopathy with cytoplasmic and nuclear inclusions in a saudi family due to dnajb6 mutation
topic Original Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6390114/
https://www.ncbi.nlm.nih.gov/pubmed/30838352
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