Myoclonus-Dystonia (M-D) is a rare autosomal-dominant movement disorder characterized by myoclonic jerks in combination with dystonia and psychiatric features. Mutations in the Epsilon-sarcoglycan (SGCE, DYT11) gene have been found to cause M-D in 30%-50% of familial M-D. Sporadic cases have also been reported. The aim of study was to investigate whether the M-D phenotype is associated with the existence of SGCE mutations in Moroccan sporadic patients with M-D syndrome. The study included 12 M-D patients. We sequenced the entire coding region of the SGCE gene. We identified two different heterozygous SGCE mutations (c.769A > C ; c.391-3T > C). Our finding confirm that SGCE mutations can occur in sporadic patients when the phenotype is consistent with M-D. Further functional studies are needed to show how changes in SGCE protein function lead to the M-D phenotype.
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http://dx.doi.org/10.1016/j.neulet.2019.03.003 | DOI Listing |
Discov Oncol
November 2024
Affiliated Stomatology Hospital of Guilin Medical University, Guilin, 541004, Guangxi, People's Republic of China.
Background: S100A8/A9, an innate immune protein, significantly regulates inflammatory processes and immune responses. While S100A8/A9 has been linked to various diseases, its association with head and neck squamous cell carcinoma (HNSCC) remains unclear.
Methods: Samples from the Cancer Genome Atlas (TCGA) were categorized into groups with low and high expression of S100A8/A9.
Cells
September 2024
Neuroscience and Mental Health Innovation Institute, Cardiff University, Hadyn Ellis Building, Maindy Road, Cardiff CF24 4HQ, UK.
Dystonia is a movement disorder with an estimated prevalence of 1.2% and is characterised by involuntary muscle contractions leading to abnormal postures and pain. Only symptomatic treatments are available with no disease-modifying or curative therapy, in large part due to the limited understanding of the underlying pathophysiology.
View Article and Find Full Text PDFMol Biol Rep
September 2024
Centogene GmbH, Rostock, Germany.
Background: Hypophosphatasia (HPP) is a rare disease caused by deficient activity of tissue-nonspecific alkaline phosphatase (ALP), encoded by the ALPL gene. The primary objective was to explore novel ALPL variants by whole genome sequencing (WGS) in patients with HPP who previously tested negative by standard methods for ALPL variants. The secondary objective was to search for genes beyond ALPL that may reduce ALP activity or contribute to HPP symptoms.
View Article and Find Full Text PDFMyoclonus-dystonia syndrome (MDS) presents with both rapid myoclonus and dystonia, which is caused by mutations in the sarcoglycan (SGCE) gene. However, its complications and management remain unclear. Here, we report a case involving a girl with MDS due to a 7q21.
View Article and Find Full Text PDFJ Neurol
June 2024
Department of Neurology, Tokyo Metropolitan Neurological Hospital, 2-6-1 Musashidai, Fuchu, Tokyo, Japan.
Background: Pallidal deep brain stimulation (GPi-DBS) is effective for treating myoclonus and dystonia caused by SGCE mutations (DYT-SGCE, DYT11). However, it is unknown whether GPi-DBS is effective for the treatment of myoclonus-dystonia which is not associated with the SGCE gene mutations. In this study, we investigated the efficacy of GPi-DBS in treating myoclonus-dystonia in SGCE mutation-negative cases.
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