CEDNIK syndrome in a Brazilian patient with compound heterozygous pathogenic variants

Eur J Med Genet. 2022 Mar;65(3):104440. doi: 10.1016/j.ejmg.2022.104440. Epub 2022 Jan 29.

Abstract

CEDNIK (Cerebral Dysgenesis, Neuropathy, Ichthyosis, and Keratoderma) syndrome is a neuro ichthyotic syndrome characterized by a clinical constellation of features including severe developmental delay, microcephaly, and facial dysmorphism. Here, we report the clinical and molecular characterization of a patient with CEDNIK syndrome harboring two compound heterozygous variants in the SNAP29 gene. The patient presents a combination of a loss-of-function SNAP29 mutation and a ∼370 kb 22q11.2 deletion, each of these genetic variants inherited from one of the parents. This report provides detailed data of a patient with unprecedented genetic events leading to the CEDNIK phenotype and may contribute to the elucidation of this rare condition.

Keywords: CEDNIK syndrome; Cerebral dysgenesis; Deletion; Ichthyosis; Keratoderma; Mutation; Neuropathy.

MeSH terms

  • Brazil
  • Humans
  • Keratoderma, Palmoplantar* / genetics
  • Keratoderma, Palmoplantar* / pathology
  • Mutation
  • Neurocutaneous Syndromes
  • Phenotype
  • Qb-SNARE Proteins / genetics
  • Qc-SNARE Proteins* / genetics

Substances

  • Qb-SNARE Proteins
  • Qc-SNARE Proteins

Supplementary concepts

  • Cerebral dysgenesis, neuropathy, ichthyosis, and palmoplantar keratoderma syndrome