Type 1 FSHD with 6-10 Repeated Units: Factors Underlying Severity in Index Cases and Disease Penetrance in Their Relatives Attention.


Journal

International journal of molecular sciences
ISSN: 1422-0067
Titre abrégé: Int J Mol Sci
Pays: Switzerland
ID NLM: 101092791

Informations de publication

Date de publication:
23 Mar 2020
Historique:
received: 27 01 2020
revised: 20 03 2020
accepted: 21 03 2020
entrez: 27 3 2020
pubmed: 27 3 2020
medline: 15 12 2020
Statut: epublish

Résumé

Molecular defects in type 1 facioscapulohumeral muscular dystrophy (FSHD) are caused by a heterozygous contraction of the D4Z4 repeat array from 1 to 10 repeat units (RUs) on 4q35. This study compared (1) the phenotype and severity of FSHD1 between patients carrying 6-8 vs. 9-10 RUs, (2) the amount of methylation in different D4Z4 regions between patients with FSHD1 with different clinical severity scores (CSS). This cross-sectional multicenter study was conducted to measure functional scales and for genetic analysis. Patients were classified into two categories according to RUs: Group 1, 6-8; Group 2, 9-10. Methylation analysis was performed in 27 patients. A total of 99 carriers of a contracted D4Z4 array were examined. No significant correlations between RUs and CSS (r = 0.04,

Identifiants

pubmed: 32210100
pii: ijms21062221
doi: 10.3390/ijms21062221
pmc: PMC7139460
pii:
doi:

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Subventions

Organisme : Association Française contre les Myopathies
ID : 10906

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Auteurs

Emmanuelle Salort-Campana (E)

Reference Center of Neuromuscular disorders and ALS, Timone University Hospital, AP-HM, 264 rue Saint-Pierre, Cedex 05 13385 Marseille, France.
Medical Genetics, Aix Marseille Université-Inserm UMR_1251, 13005 Marseille, France.

Farzad Fatehi (F)

Reference Center of Neuromuscular disorders and ALS, Timone University Hospital, AP-HM, 264 rue Saint-Pierre, Cedex 05 13385 Marseille, France.

Sadia Beloribi-Djefaflia (S)

Reference Center of Neuromuscular disorders and ALS, Timone University Hospital, AP-HM, 264 rue Saint-Pierre, Cedex 05 13385 Marseille, France.

Stéphane Roche (S)

Medical Genetics, Aix Marseille Université-Inserm UMR_1251, 13005 Marseille, France.

Karine Nguyen (K)

Medical Genetics, Aix Marseille Université-Inserm UMR_1251, 13005 Marseille, France.

Rafaelle Bernard (R)

Medical Genetics, Aix Marseille Université-Inserm UMR_1251, 13005 Marseille, France.

Pascal Cintas (P)

Service de Neurologie et d'explorations fonctionnelles, Centre Hospitalier Universitaire de Toulouse, 31000 Toulouse, France.

Guilhem Solé (G)

Reference Center of Neuromuscular Disorders AOC, Bordeaux University Hospitals, 33000 Bordeaux, France.

Françoise Bouhour (F)

Electroneuromyography and Neuromuscular Department, GHE Neurologic Hospital, Cedex 69677 Lyon-Bron, France.

Elisabeth Ollagnon (E)

Neurogenetic Department, GHN Croix-Rousse Hospital, 69004 Lyon, France.

Sabrina Sacconi (S)

Neuromuscular Disease Specialized Center, Nice University Hospital, 06000 Nice, France.

Andoni Echaniz-Laguna (A)

Neurology Department, APHP, CHU de Bicêtre, 78 rue du Général Leclerc, Cedex 94276 Le Kremlin-Bicêtre, France.

Thierry Kuntzer (T)

Nerve-Muscle Unit, Department of Clinical Neurosciences, Lausanne University, Hospital (CHUV), Lausanne 1002, Switzerland.

Nicolas Levy (N)

Medical Genetics, Aix Marseille Université-Inserm UMR_1251, 13005 Marseille, France.

Frédérique Magdinier (F)

Medical Genetics, Aix Marseille Université-Inserm UMR_1251, 13005 Marseille, France.

Shahram Attarian (S)

Reference Center of Neuromuscular disorders and ALS, Timone University Hospital, AP-HM, 264 rue Saint-Pierre, Cedex 05 13385 Marseille, France.
Medical Genetics, Aix Marseille Université-Inserm UMR_1251, 13005 Marseille, France.

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