| Literature DB >> 32700429 |
Nicole Nakousi-Capurro1, Jonathan Huserman1, Silvia Castillo1,2, Luisa Herrera3, Pablo Romero4, Felipe Pizarro5, Cristian Quezada5, Francisco Cea6.
Abstract
Knobloch Syndrome (KS) is a rare autosomal recessive hereditary disease. Despite its clinical heterogeneity, it is characterized by vitreoretinal degeneration and high myopia, with or without occipital skull defects. It is caused by mutations in the COL18A1 gene, which codifies for collagen XVIII, present in retina and vascular endothelium. Since the first description of the disease by doctors Knobloch and Layer in 1972, over 100 cases and 20 pathogenic or likely pathogenic mutations have been reported. We present the case of a child born from a consanguineous couple in Chile with congenital high myopia and dysmorphisms without an occipital skull defect. Whole exome sequencing analysis revealed an inherited homozygous variant in COL18A1, c.4224_4225delinsC, p.Pro1411Leufs*35.Entities:
Keywords: COL18A1; Knobloch syndrome; high-grade myopia
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Year: 2020 PMID: 32700429 DOI: 10.1002/ajmg.a.61760
Source DB: PubMed Journal: Am J Med Genet A ISSN: 1552-4825 Impact factor: 2.802