[NEXT-GENERATION SEQUENCING PERFORMED IN PATIENTS RAISING THE SUSPICION OF AN INBORN ERROR OF METABOLISM UNCOVERED A HOMOZYGOUS VARIANT IN YARS1 ALLOWING A NOVEL THERAPEUTIC TRIAL]

Harefuah. 2023 Jun;162(6):344-351.
[Article in Hebrew]

Abstract

Inborn-Errors of Metabolism (IEM) are genetic disorders resulting from mutations in genes encoding proteins involved in biochemical-metabolic pathways. However, some IEMs lack specific biochemical markers. Early incorporation of next-generation-sequencing (NGS) including whole exome sequencing (WES) into the diagnostic algorithm of IEMs herein provided, increases diagnostic accuracy, permits genetic counseling and improves therapeutic options. This is exemplified by diseases affecting aminoacyl-tRNA synthetases (ARSs), enzymes involved in protein translation. Recent studies showed that supplementing amino-acids to cell-culture and patients with ARSs deficiencies resulted in improvement of biochemical and clinical parameters, respectively.

Publication types

  • English Abstract

MeSH terms

  • Biomarkers
  • Genetic Counseling
  • High-Throughput Nucleotide Sequencing / methods
  • Humans
  • Metabolism, Inborn Errors* / diagnosis
  • Metabolism, Inborn Errors* / genetics
  • Metabolism, Inborn Errors* / therapy
  • Mutation

Substances

  • Biomarkers