Congenital disorders of glycosylation (CDGs) are a genetically heterogeneous group of disorders caused by the defects in the synthesis and processing of glycoproteins. CDG is caused by mannosyl-oligosaccharide glucosidase (MOGS) deficiency, and is an extremely rare type, and only six patients have been reported. Here, we report a patient from China with facial dysmorphism, infantile spams, developmental delay, low vison, and abnormal liver function and low immunoglobulin. Brain MRI showed hypoplasia of the corpus callosum and slightly wide sulci at bilateral frontal parietal lobes. Compound heterozygous mutations of (c.1694G>A: R565Q and c.1619G>A: R540H) in exon 4 of MOGS gene (NM_006302.2) were identified by whole exome sequencing. Further investigation showed that the gene expression of MOGS in patients' peripheral blood was decreased. We observed that two mutations were associated with lower protein expression of MOGS, cell growth, and cell cycle in transiently transfected Hela cells. We also noticed that cell cycle-related proteins, β-catenin, cyclin D1, and C-myc, were decreased in mutant cells. In conclusion, our study suggested whole exome sequencing, and genes associated with CDGs should be analyzed in patients with infantile spams and multiple system involvement, and mutant MOGS-impaired cell cycle progression. Our work broadens the mutation spectrum of MOGS gene.
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http://dx.doi.org/10.1007/s10048-019-00590-5 | DOI Listing |
Epilepsia
September 2023
Rare Brain Disorders Program, Department of Neurology, The University of Texas Southwestern Medical Center, Dallas, Texas, USA.
Epilepsy constitutes the most common paroxysmal manifestation of glucose transporter type 1 deficiency (G1D) and is generally considered medication-refractory. It can also prove therapeutic diet-resistant. We examined acetazolamide effects in G1D motivated by several longstanding and recent observations: First, the electrographic spike-waves characteristic of absence seizures often resemble those of G1D and, since the 1950s, they have occasionally been treated successfully with acetazolamide, well before G1D was segregated from absence epilepsy as a distinct syndrome.
View Article and Find Full Text PDFEpilepsy Res
February 2023
Department of Neurology, Hospital de Padiatría J.P. Garrahan, Buenos Aires, Argentina. Electronic address:
Objective: The study was conducted to analyze the possible diagnostic value of the electroclinical semiology of the epileptic seizures.
Methods: We evaluated the medical records of 17 females and 5 males with CDKL5 deficiency disorder (CDD) considering the long-term evolution, including the polygraphic video-EEG recordings.
Results: We recognized three disease phases.
Epilepsia Open
December 2021
Division of Epilepsy and Clinical Neurophysiology, Department of Neurology, Boston Children's Hospital, Boston, Massachusetts, USA.
Objective: There is no consensus on the type or duration of the posttreatment EEG needed for assessing treatment response for infantile spasms (IS). We assessed whether outpatient electroencephalograms (EEGs) are sufficient to confirm infantile spasms (IS) treatment response.
Methods: Three-year retrospective review identified new-onset IS patients.
Pediatr Neurol
September 2021
MD Program, Weill Cornell Medicine, New York, New York; Department of Pediatrics, Weill Cornell Medicine, New York, New York; Department of Population Health Sciences, Weill Cornell Medicine, New York, New York. Electronic address:
Background: Use of electronic seizure diaries (e-diaries) by caregivers of children with epileptic spasms is not well understood. We describe the demographic and seizure-related information of children with epileptic spasms captured in a widely used e-diary and explore the potential biases in how caregivers report these data.
Methods: We analyzed children with epileptic spasms in an e-diary, Seizure Tracker, from 2007 to 2018.
Gene
August 2020
Institute of Human Genetics, Klinikum rechts der Isar, Technical University of Munich, School of Medicine, Munich, Germany. Electronic address:
Lymphedema are characterized by interstitial edema leading to swelling of extremities. They can be divided into primary and secondary lymphedema. Developmental abnormalities of the lymphatic system are responsible for the primary form of lymphedema.
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