Whole exome sequencing revealed novel variants in consanguineous Pakistani families with intellectual disability

Genes Genomics. 2021 May;43(5):503-512. doi: 10.1007/s13258-021-01070-7. Epub 2021 Mar 12.

Abstract

Background: Intellectual disability (ID) is a heterogeneous disorder affecting 1-3% of the population. Elucidation of monogenic variants for ID is a current challenge. These variants can be better demonstrated in consanguineous affected families.

Objective: The study was designed to find the genetic variants of ID in consanguineous families.

Methods: We analyzed five unrelated consanguineous Pakistani families affected with ID using whole exome sequencing (WES). Data was analyzed using different bioinformatics tools and software.

Results: We mapped four variants including three novels in four different ID known genes. Each variant is found in a different family, co-segregating with a recessive pattern of inheritance. The novel variants found are; c. 2_4del (p.?) mapped in ROS1 and c. 718G>A (p.Gly240Arg) in GRM1. Another novel causative variant, c.2673del (p.Gly892Aspfs*17) identified in COL18A1 in a recessive form, a gene reported for Knobloch syndrome that manifests ID along with typical retinal abnormalities, and this phenotype was confirmed on reverse phenotyping. A mutation c.2134C>T (p.Arg712*) in TRAPPC9 has been found first time in the homozygous recessive form in our enrolled three affected siblings while it was previously reported in compound heterozygous form in a Caucasian descent. While fifth family remained unsolved.

Conclusion: These mutations in four different genes with a recessive inheritance would be a contribution to the disease variant database of this devastating disorder.

Keywords: Consanguineous; Intellectual disability; Mutations; Pakistani population; Whole exome sequencing.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Child
  • Collagen Type XVIII / genetics
  • Consanguinity
  • Exome Sequencing
  • Female
  • Humans
  • Intellectual Disability / genetics*
  • Intellectual Disability / pathology
  • Intercellular Signaling Peptides and Proteins / genetics
  • Male
  • Mutation*
  • Pedigree
  • Phenotype
  • Protein-Tyrosine Kinases / genetics
  • Proto-Oncogene Proteins / genetics
  • Receptors, Metabotropic Glutamate / genetics

Substances

  • COL18A1 protein, human
  • Collagen Type XVIII
  • GRM1 protein, human
  • Intercellular Signaling Peptides and Proteins
  • Proto-Oncogene Proteins
  • Receptors, Metabotropic Glutamate
  • TRAPPC9 protein, human
  • Protein-Tyrosine Kinases
  • ROS1 protein, human