SCAF4-related syndromic intellectual disability |
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Authors: | Laura Machado Lara Carvalho Carla Franchi Pinto Marília de Oliveira Scliar Paulo A. Otto Ana Cristina Victorino Krepischi Carla Rosenberg |
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Affiliation: | 1. Human Genome and Stem Cell Research Center, Department of Genetics and Evolutionary Biology, Institute of Biosciences, University of Sao Paulo (USP), Sao Paulo, Sao Paulo, Brazil;2. Department of Pathological Sciences, Faculty of Medical Sciences of Santa Casa de São Paulo, Sao Paulo, Sao Paulo, Brazil |
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Abstract: | The causal link between variants in the SCAF4 gene and a syndromic form of intellectual disability (ID) was established in 2020 by Fliedner et al. Since then, no additional cases have been reported. We performed exome sequencing in a 16-year-old Brazilian male presenting with ID, epilepsy, behavioral problems, speech impairment, facial dysmorphisms, heart malformations, and obesity. A de novo pathogenic variant [SCAF4(NM_020706.2):c.374_375dup(p.Glu126LeufsTer20)] was identified. This is the second study reporting the involvement of SCAF4 in syndromic ID, and the description of the patient's clinical features contributes to defining the phenotypic spectrum of this recently described Mendelian disorder. |
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Keywords: | epilepsy exome sequencing human genetics intellectual disability obesity SCAF4 |
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