Female-restricted syndromic intellectual disability in a patient from Thailand.
Thivaratana SinthuwiwatChupong IttiwutThantrira PorntaveetusVorasuk ShotelersukPublished in: American journal of medical genetics. Part A (2019)
Female-restricted syndromic intellectual disability (ID) is a neurodevelopmental disorder with developmental delay (DD)/ID, facial dysmorphism, and diverse congenital anomalies comprising heart defects, anal anomalies, choanal atresia, postaxial polydactyly, scoliosis, and brain abnormalities. Loss-of-function mutations in the USP9X gene inherited as X-linked dominance were identified as its etiology in females of different ethnic groups. Here, we report a 15-year-old Thai girl harboring a novel de novo heterozygous one-base pair deletion (c.3508delG, p.Val1170TrpfsX9) in exon 23 of USP9X. Her profound DD, dysmorphic face including attached earlobes, short stature, and congenital malformations including s-shaped thoracolumbar scoliosis, hip dislocation, and generalized brain atrophy shared common characteristics of X-linked syndromic ID. We have observed severely malformed oro-dental organs and a choledochal cyst, which have never been reported. Our study presents the first patient from Thailand expanding the phenotypic and mutational spectra of the syndrome.
Keyphrases
- intellectual disability
- autism spectrum disorder
- case report
- resting state
- white matter
- heart failure
- functional connectivity
- early onset
- cerebral ischemia
- high grade
- atrial fibrillation
- copy number
- genome wide
- density functional theory
- dna methylation
- blood brain barrier
- genome wide identification
- molecular dynamics