Autistic behavior is a common outcome of biallelic disruption of PDZD8 in humans and mice.

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Tác giả: Serpil Alkan, Christophe Barrea, Camille Chatelain, Steven J Clapcote, Elfride De Baere, Beatrice M Filippi, Jamie Johnston, Jason P Lerch, Clemence Ligneul, Andreea D Pantiru, Stijn Van de Sompele

Ngôn ngữ: eng

Ký hiệu phân loại: 272.3 Persecutions of Waldenses and Albigenses

Thông tin xuất bản: England : Molecular autism , 2025

Mô tả vật lý:

Bộ sưu tập: NCBI

ID: 682316

BACKGROUND: Intellectual developmental disorder with autism and dysmorphic facies (IDDADF) is a rare syndromic intellectual disability (ID) caused by homozygous disruption of PDZD8 (PDZ domain-containing protein 8), an integral endoplasmic reticulum (ER) protein. All four previously identified IDDADF cases exhibit autistic behavior, with autism spectrum disorder (ASD) diagnosed in three cases. To determine whether autistic behavior is a common outcome of PDZD8 disruption, we studied a third family with biallelic mutation of PDZD8 (family C) and further characterized PDZD8-deficient (Pdzd8 METHODS: Homozygosity mapping, whole-exome sequencing, and cosegregation analysis were used to identify the PDZD8 variant responsible for IDDADF, including diagnoses of ASD, in consanguineous family C. To assess the in vivo effect of PDZD8 disruption on social responses and related phenotypes, behavioral, structural magnetic resonance imaging, and microscopy analyses were conducted on the Pdzd8 RESULTS: The discovery of a third family with IDDADF caused by biallelic disruption of PDZD8 permitted identification of a core clinical phenotype consisting of developmental delay, ID, autism, and facial dysmorphism. In addition to impairments in social recognition and social odor discrimination, Pdzd8 LIMITATIONS: The total number of known cases of PDZD8-related IDDADF remains low. Some mouse experiments in the study did not use balanced numbers of males and females. The assessment of ER stress and mitochondrial fusion markers did not extend beyond mRNA levels. CONCLUSIONS: Our finding that the Pdzd8
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