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DOI 10.1080/01677063.2018.1476510

Медицинская библиотека · Journal of neurogenetics · 2018

A novel SLC1A4 homozygous mutation causing congenital microcephaly, epileptic encephalopathy and spastic tetraparesis: a video-EEG and tractography - case study.

Pironti Erica, Salpietro Vincenzo, Cucinotta Francesca, Granata Francesca, Mormina Enricomaria, Efthymiou Stephanie, Scuderi Carmela, Gagliano Antonella, Houlden Henry, Di Rosa Gabriella

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Аннотация

Biallelic mutations in the SLC1A4 gene have been identified as a very rare cause of neurodevelopmental disorders. l-serine transport deficiency has been regarded as the causal molecular mechanism underlying the neurological phenotype of SLC1A4 mutation patients. To date this genetic condition has been reported almost exclusively in a limited number of Ashkenazi-Jewish individuals and as a result the SLC1A4 gene is not routinely included in the majority of the genetic diagnostic panels for neurological diseases. We hereby report a 7-year-old boy from a Southern Italian family, presenting with epileptic encephalopathy, congenital microcephaly, global developmental delay, severe hypotonia, spasticity predominant at the lower limbs, and thin corpus callosum. Whole exome sequencing identified a novel segregating SLC1A4 gene homozygous mutation (c.1141G > A: p.Gly381Arg) as the likely cause of the disease in our family. In order to deeply characterize the electro-clinical and neurological phenotype in our index patient, long-term systematic video-electroencephalograms (EEG) as well as repeated brain imaging studies (which included tractographic reconstructions) were performed on a regular basis during a 7 years follow-up time. In conclusion, we suggest to carefully considering SLC1A4 biallelic mutations in individuals presenting an early onset severe neurodevelopmental disorder with variable spasticity and seizures, regardless the patients' ethnic background.

Для цитирования: Pironti Erica, Salpietro Vincenzo, Cucinotta Francesca, Granata Francesca, Mormina Enricomaria, Efthymiou Stephanie, Scuderi Carmela, Gagliano Antonella, Houlden Henry, Di Rosa Gabriella. A novel SLC1A4 homozygous mutation causing congenital microcephaly, epileptic encephalopathy and spastic tetraparesis: a video-EEG and tractography - case study.. Journal of neurogenetics. 2018. DOI: 10.1080/01677063.2018.1476510.

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