Primary adhalinopathy: a common cause of autosomal recessive muscular dystrophy of variable severity.

Détails

ID Serval
serval:BIB_BDEE10417BC6
Type
Article: article d'un périodique ou d'un magazine.
Collection
Publications
Institution
Titre
Primary adhalinopathy: a common cause of autosomal recessive muscular dystrophy of variable severity.
Périodique
Nature Genetics
Auteur⸱e⸱s
Piccolo F., Roberds S.L., Jeanpierre M., Leturcq F., Azibi K., Beldjord C., Carrié A., Récan D., Chaouch M., Reghis A., Elkerch F., Sefiani A., Voit T., Merlini L., Collin H., Eymard B., Beckmann J.S., Romero N.B., Tomé F.M.S., Fardeau M., Campbell K.P., Kaplan C
ISSN
1061-4036
Statut éditorial
Publié
Date de publication
1995
Peer-reviewed
Oui
Volume
10
Numéro
2
Pages
243-245
Langue
anglais
Notes
Publication types: Journal Article ; Research Support, Non-U.S. Gov't - Publication Status: ppublish
Résumé
Marked deficiency of muscle adhalin, a 50 kDa sarcolemmal dystrophin-associated glycoprotein, has been reported in severe childhood autosomal recessive muscular dystrophy (SCARMD). This is a Duchenne-like disease affecting both males and females first described in Tunisian families. Adhalin deficiency has been found in SCARMD patients from North Africa Europe, Brazil, Japan and North America (SLR & KPC, unpublished data). The disease was initially linked to an unidentified gene on chromosome 13 in families from North Africa, and to the adhalin gene itself on chromosome 17q in one French family in which missense mutations were identified. Thus there are two kinds of myopathies with adhalin deficiency: one with a primary defect of adhalin (primary adhalinopathies), and one in which absence of adhalin is secondary to a separate gene defect on chromosome 13. We have examined the importance of primary adhalinopathies among myopathies with adhalin deficiency, and describe several additional mutations (null and missense) in the adhalin gene in 10 new families from Europe and North Africa. Disease severity varies in age of onset and rate of progression, and patients with null mutations are the most severely affected.
Mots-clé
Adolescent, Base Sequence, Child, Child, Preschool, Cytoskeletal Proteins/analysis, Cytoskeletal Proteins/deficiency, Dystrophin/analysis, Dystrophin/genetics, Female, Genes, Recessive, Humans, Male, Membrane Glycoproteins/analysis, Membrane Glycoproteins/deficiency, Models, Molecular, Molecular Sequence Data, Muscular Dystrophies/genetics, Point Mutation, Protein Conformation, Sarcoglycans, Severity of Illness Index
Pubmed
Web of science
Création de la notice
25/01/2008 17:17
Dernière modification de la notice
20/08/2019 16:32
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