Targeted next generation sequencing reveals genetic defects underlying inherited retinal disease in Iranian families

Mol Vis. 2019 Feb 8:25:106-117. eCollection 2019.

Abstract

Purpose: Inherited retinal diseases (IRDs) are clinically and genetically heterogeneous showing progressive retinal cell death which results in vision loss. IRDs include a wide spectrum of disorders, such as retinitis pigmentosa (RP), Leber congenital amaurosis (LCA), cone-rod dystrophy (CRD), and Stargardt disease (STGD1).

Methods: In this study, we performed targeted next-generation sequencing based on molecular inversion probes (MIPs) that allowed the sequence analysis of 108 IRD-associated genes in 50 Iranian IRD probands.

Results: The sequencing and variant filtering led to the identification of putative pathogenic variants in 36 out of 50 (72%) probands. Among 36 unique variants, we identified 20 novel variants in 15 genes. Four out of 36 probands carry compound heterozygous variants, and 32 probands carry homozygous variants.

Conclusions: Employing a cost-effective targeted next-generation sequencing procedure, we identified the genetic causes of different retinal disorders in the majority of Iranian families in this study.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Child
  • Cone-Rod Dystrophies / genetics*
  • Cone-Rod Dystrophies / metabolism
  • Cone-Rod Dystrophies / pathology
  • Eye Proteins / genetics*
  • Eye Proteins / metabolism
  • Female
  • Gene Expression
  • Genetic Association Studies
  • Genotype
  • Heterozygote
  • High-Throughput Nucleotide Sequencing / economics
  • High-Throughput Nucleotide Sequencing / methods
  • Homozygote
  • Humans
  • Iran
  • Leber Congenital Amaurosis / genetics*
  • Leber Congenital Amaurosis / metabolism
  • Leber Congenital Amaurosis / pathology
  • Macular Degeneration / congenital*
  • Macular Degeneration / genetics
  • Macular Degeneration / metabolism
  • Macular Degeneration / pathology
  • Male
  • Mutation*
  • Pedigree
  • Phenotype
  • Retina / metabolism
  • Retina / pathology
  • Retinitis Pigmentosa / congenital
  • Retinitis Pigmentosa / genetics*
  • Retinitis Pigmentosa / metabolism
  • Retinitis Pigmentosa / pathology
  • Stargardt Disease

Substances

  • Eye Proteins