Chung-Jansen syndrome can mimic Cornelia de Lange syndrome: Another player among chromatinopathies?
Beatrice ContiBerardo RinaldiMartina RimoldiRoberta VillaMaria IasconeSilvana GangiMatteo PorroPaola Francesca AjmoneAnna Maria ColliFabio MoscaMaria Francesca BedeschiPublished in: American journal of medical genetics. Part A (2023)
Cornelia de Lange syndrome (CdLS) is a rare multisystem congenital neurodevelopmental disorder (NDD) characterized by distinctive facial anomalies, short stature, developmental delay, hirsutism, gastrointestinal abnormalities and upper limb reduction defects. CdLS syndrome is associated with causative variants in genes encoding for the cohesin complex, a cellular machinery involved in chromatid pairing, DNA repair and gene-expression regulation. In this report, we describe a familial case of a syndromic presentation in a 4-year-old patient (P1) and in his mother (P2). Trio-based Whole Exome Sequencing (WES) performed on P1 was first negative. Since his phenotypic evolution during the follow-up was reminiscent of the CdLS spectrum, a reanalysis of WES data, focused on CdLS-related genes, was requested. Although no alterations in those genes was detected, we identified the likely pathogenetic variant c.40G > A (p.Glu14Lys) in the PHIP gene, in the meanwhile associated with Chung-Jansen syndrome. Reverse phenotyping carried out in both patients confirmed the molecular diagnosis. CHUJANS belongs to NDDs, featuring developmental delay, mild-to-moderate intellectual disability, behavioral problems, obesity and facial dysmorphisms. Moreover, as here described, CHUJANS shows a significant overlap with the CdLS spectrum, with specific regard to facial gestalt. On the basis of our findings, we suggest to include PHIP among genes routinely analyzed in patients belonging to the CdLS spectrum.
Keyphrases
- intellectual disability
- case report
- dna repair
- gene expression
- end stage renal disease
- genome wide
- ejection fraction
- newly diagnosed
- upper limb
- metabolic syndrome
- autism spectrum disorder
- peritoneal dialysis
- type diabetes
- prognostic factors
- copy number
- insulin resistance
- soft tissue
- dna methylation
- machine learning
- dna damage response
- body mass index
- oxidative stress
- adipose tissue
- patient reported