Literature DB >> 11222786

Autosomal dominant limb-girdle muscular dystrophy: a large kindred with evidence for anticipation.

J Gamez1, C Navarro, A L Andreu, J M Fernandez, L Palenzuela, S Tejeira, R Fernandez-Hojas, S Schwartz, C Karadimas, S DiMauro, M Hirano, C Cervera.   

Abstract

BACKGROUND: Fourteen genetically distinct forms of limb-girdle muscular dystrophy (LGMD) have been identified, including five types of autosomal dominant LGMD (AD-LGMD).
OBJECTIVE: To describe clinical, histologic, and genetic features of a large Spanish kindred with LGMD and apparent autosomal dominant inheritance spanning five generations.
METHOD: The authors examined 61 members of the family; muscle biopsies were performed on five patients. Linkage analysis assessed chromosomal loci associated with other forms of AD-LGMD.
RESULTS: A total of 32 individuals had weakness of the pelvic and shoulder girdles. Severity appeared to worsen in successive generations. Muscle biopsy findings were nonspecific and compatible with MD. Linkage analysis to chromosomes 5q31, 1q11-q21, 3p25, 6q23, and 7q demonstrated that this disease is not allelic to LGMD forms 1A, 1B, 1C, 1D, and 1E.
CONCLUSIONS: This family has a genetically distinct form of AD-LGMD. The authors are currently performing a genome-wide scan to identify the disease locus.

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Year:  2001        PMID: 11222786     DOI: 10.1212/wnl.56.4.450

Source DB:  PubMed          Journal:  Neurology        ISSN: 0028-3878            Impact factor:   9.910


  14 in total

1.  A new locus on 3p23-p25 for an autosomal-dominant limb-girdle muscular dystrophy, LGMD1H.

Authors:  Luigi Bisceglia; Stefano Zoccolella; Alessandra Torraco; Maria Rosaria Piemontese; Rosa Dell'Aglio; Angela Amati; Patrizia De Bonis; Lucia Artuso; Massimiliano Copetti; Filippo Maria Santorelli; Luigi Serlenga; Leopoldo Zelante; Enrico Bertini; Vittoria Petruzzella
Journal:  Eur J Hum Genet       Date:  2010-01-13       Impact factor: 4.246

2.  A mutation in the dimerization domain of filamin c causes a novel type of autosomal dominant myofibrillar myopathy.

Authors:  Matthias Vorgerd; Peter F M van der Ven; Vera Bruchertseifer; Thomas Löwe; Rudolf A Kley; Rolf Schröder; Hanns Lochmüller; Mirko Himmel; Katrin Koehler; Dieter O Fürst; Angela Huebner
Journal:  Am J Hum Genet       Date:  2005-05-31       Impact factor: 11.025

3.  Limb-girdle muscular dystrophy 1F is caused by a microdeletion in the transportin 3 gene.

Authors:  Maria J Melià; Akatsuki Kubota; Saida Ortolano; Juan J Vílchez; Josep Gámez; Kurenai Tanji; Eduardo Bonilla; Lluís Palenzuela; Israel Fernández-Cadenas; Anna Pristoupilová; Elena García-Arumí; Antoni L Andreu; Carmen Navarro; Michio Hirano; Ramon Martí
Journal:  Brain       Date:  2013-03-29       Impact factor: 13.501

4.  Exome sequencing reveals DNAJB6 mutations in dominantly-inherited myopathy.

Authors:  Matthew B Harms; R Brian Sommerville; Peggy Allred; Shaughn Bell; Duanduan Ma; Paul Cooper; Glenn Lopate; Alan Pestronk; Conrad C Weihl; Robert H Baloh
Journal:  Ann Neurol       Date:  2012-02-14       Impact factor: 10.422

5.  Muscle phenotypic variability in limb girdle muscular dystrophy 2 G.

Authors:  Julia F Paim; Ana Cotta; Antonio P Vargas; Monica M Navarro; Jaquelin Valicek; Elmano Carvalho; Antonio L da-Cunha; Estevão Plentz; Shelida V Braz; Reinaldo I Takata; Camila F Almeida; Mariz Vainzof
Journal:  J Mol Neurosci       Date:  2013-03-12       Impact factor: 3.444

6.  Clinical phenotype, muscle MRI and muscle pathology of LGMD1F.

Authors:  Enrico Peterle; Marina Fanin; Claudio Semplicini; Juan Jesus Vilchez Padilla; Vincenzo Nigro; Corrado Angelini
Journal:  J Neurol       Date:  2013-04-30       Impact factor: 4.849

7.  The Classification, Natural History and Treatment of the Limb Girdle Muscular Dystrophies.

Authors:  Alexander Peter Murphy; Volker Straub
Journal:  J Neuromuscul Dis       Date:  2015-07-22

8.  Long term follow-up and further molecular and histopathological studies in the LGMD1F sporadic TNPO3-mutated patient.

Authors:  Sara Gibertini; Alessandra Ruggieri; Simona Saredi; Franco Salerno; Flavia Blasevich; Laura Napoli; Maurizio Moggio; Vincenzo Nigro; Lucia Morandi; Lorenzo Maggi; Marina Mora
Journal:  Acta Neuropathol Commun       Date:  2018-12-19       Impact factor: 7.801

9.  Novel mutation in TNPO3 causes congenital limb-girdle myopathy with slow progression.

Authors:  Anna Vihola; Johanna Palmio; Olof Danielsson; Sini Penttilä; Daniel Louiselle; Sara Pittman; Conrad Weihl; Bjarne Udd
Journal:  Neurol Genet       Date:  2019-05-02

10.  Next-generation sequencing identifies transportin 3 as the causative gene for LGMD1F.

Authors:  Annalaura Torella; Marina Fanin; Margherita Mutarelli; Enrico Peterle; Francesca Del Vecchio Blanco; Rossella Rispoli; Marco Savarese; Arcomaria Garofalo; Giulio Piluso; Lucia Morandi; Giulia Ricci; Gabriele Siciliano; Corrado Angelini; Vincenzo Nigro
Journal:  PLoS One       Date:  2013-05-07       Impact factor: 3.240

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