Literature DB >> 26733286

Distal acroosteolysis, poikiloderma and joint stiffness: a novel laminopathy?

Wafaa Sewairi1, Abdulrahman Assiri2, Nisha Patel3, Amal Alhashem2,4, Fowzan S Alkuraya3,4.   

Abstract

LMNA encodes lamin A and lamin C, two major components of the nuclear lamina, and its pathogenic variants lead to a dozen distinct clinical entities collectively known as laminopathies. Most LMNA-related laminopathies are autosomal dominant but four are autosomal recessive; furthermore, some of the dominant variants have been associated with distinct phenotypes when inherited recessively, further complicating the ability to correlate genotype with phenotype. We report a consanguineous family in which the index presented with an apparently unique constellation of poikiloderma, joint motion restriction and distal acroosteolysis but lacks features of muscle weakness, lipodystrophy, or cardiac or craniofacial involvement. Molecular analysis revealed the presence of a novel homozygous LMNA missense variant (NM_170707.3:c.1774G>A; p.(Gly592Arg)) within an area of autozygome that is not shared by his unaffected siblings. The proposed causal link is further supported by in silico analysis of this variant. Our case suggests an expansion of LMNA allelic disorders to include distal acroosteolysis, poikiloderma and joint stiffness (DAPJ).

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Year:  2016        PMID: 26733286      PMCID: PMC4970680          DOI: 10.1038/ejhg.2015.265

Source DB:  PubMed          Journal:  Eur J Hum Genet        ISSN: 1018-4813            Impact factor:   4.246


  18 in total

1.  Autosomal recessive LMNA mutation causing restrictive dermopathy.

Authors:  G J Youn; M Uzunyan; L Vachon; J Johnson; T L Winder; S Yano
Journal:  Clin Genet       Date:  2010-08       Impact factor: 4.438

Review 2.  Nuclear lamins and laminopathies.

Authors:  Howard J Worman
Journal:  J Pathol       Date:  2011-11-14       Impact factor: 7.996

Review 3.  Laminopathies: multisystem dystrophy syndromes.

Authors:  Katherine N Jacob; Abhimanyu Garg
Journal:  Mol Genet Metab       Date:  2005-12-20       Impact factor: 4.797

4.  cDNA sequencing of nuclear lamins A and C reveals primary and secondary structural homology to intermediate filament proteins.

Authors:  D Z Fisher; N Chaudhary; G Blobel
Journal:  Proc Natl Acad Sci U S A       Date:  1986-09       Impact factor: 11.205

5.  Homozygous defects in LMNA, encoding lamin A/C nuclear-envelope proteins, cause autosomal recessive axonal neuropathy in human (Charcot-Marie-Tooth disorder type 2) and mouse.

Authors:  Annachiara De Sandre-Giovannoli; Malika Chaouch; Serguei Kozlov; Jean-Michel Vallat; Meriem Tazir; Nadia Kassouri; Pierre Szepetowski; Tarik Hammadouche; Antoon Vandenberghe; Colin L Stewart; Djamel Grid; Nicolas Lévy
Journal:  Am J Hum Genet       Date:  2002-01-17       Impact factor: 11.025

6.  Progeria: a paradigm for translational medicine.

Authors:  Leslie B Gordon; Frank G Rothman; Carlos López-Otín; Tom Misteli
Journal:  Cell       Date:  2014-01-30       Impact factor: 41.582

Review 7.  Restrictive dermopathy--a lethal congenital laminopathy. Case report and review of the literature.

Authors:  Paulo Morais; Sofia Magina; Maria do Céu Ribeiro; Manuela Rodrigues; José Manuel Lopes; Huong Le Thi Thanh; Manfred Wehnert; Hercília Guimarães
Journal:  Eur J Pediatr       Date:  2008-11-20       Impact factor: 3.183

Review 8.  Overlapping syndromes in laminopathies: a meta-analysis of the reported literature.

Authors:  Nicola Carboni; Luisa Politano; Matteo Floris; Anna Mateddu; Elisabetta Solla; Stefania Olla; Lorenzo Maggi; Maria Antonietta Maioli; Rachele Piras; Eleonora Cocco; Giovanni Marrosu; Maria Giovanna Marrosu
Journal:  Acta Myol       Date:  2013-05

9.  Comprehensive gene panels provide advantages over clinical exome sequencing for Mendelian diseases.

Authors: 
Journal:  Genome Biol       Date:  2015-06-26       Impact factor: 13.583

10.  Mutation analysis of the lamin A/C gene (LMNA) among patients with different cardiomuscular phenotypes.

Authors:  M Vytopil; S Benedetti; E Ricci; G Galluzzi; A Dello Russo; L Merlini; G Boriani; M Gallina; L Morandi; L Politano; M Moggio; L Chiveri; I Hausmanova-Petrusewicz; R Ricotti; S Vohanka; J Toman; D Toniolo
Journal:  J Med Genet       Date:  2003-12       Impact factor: 6.318

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  4 in total

Review 1.  Discovery of mutations for Mendelian disorders.

Authors:  Fowzan S Alkuraya
Journal:  Hum Genet       Date:  2016-04-11       Impact factor: 4.132

2.  Nail changes in acro-osteolysis: A case report and review of the literature.

Authors:  Nicola Anne Gray; Christiaan Scott; Reginald Mzudumile Ngwanya; Komala Pillay; Thuraya Isaacs
Journal:  JAAD Case Rep       Date:  2019-11-13

3.  Is an association of acro-osteolysis, bone fragility, and enchondromatosis a newfound disease caused by an amplification of PTHLH? A case report.

Authors:  Stéphane Echaubard; Céline Pebrel-Richard; Aurélie Chausset; Jean-Louis Kemeny; Etienne Merlin; Fanny Laffargue
Journal:  Pediatr Rheumatol Online J       Date:  2022-07-30       Impact factor: 3.413

Review 4.  Lost bones: differential diagnosis of acro-osteolysis seen by the pediatric rheumatologist.

Authors:  Elizaveta Limenis; Jennifer Stimec; Peter Kannu; Ronald M Laxer
Journal:  Pediatr Rheumatol Online J       Date:  2021-07-14       Impact factor: 3.054

  4 in total

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