Clinical utility of Whole Exome Sequencing for rare Mendelian disorders:
phenotypic-driven strategy for a high diagnostic yield and
identification of 48 novel variants
Abstract
About 6,000 to 7,000 different rare disorders with suspected genetic
etiologies have been described and in almost 4,500 of them the causative
gene(s) have been identified. The advent of Next-Generation Sequencing
(NGS) technologies has revolutionized genomic research and diagnostics,
representing a major advance in identification of pathogenic genetic
variations. WES facilitates a faster and more cost-effective route for
definite diagnosis of rare genetic disorders, minimizing previous
“diagnostic odysseys” for the patients. Due to the limitation that WES
is not reimbursed in Greece, we aimed to minimize cost per
patient/family through applying WES in the proband, followed by targeted
family segregation studies when necessary. Furthermore, for variant
filtration and interpretation we applied a phenotypic-driven strategy in
close collaboration with clinical geneticists or referring clinicians.
In this study we report the clinical application of WES in the diagnosis
of 162 cases referred to investigate patients with undiagnosed genetic
disorders. The overall molecular diagnostic yield reached 52.5%. Our
experience as an academic diagnostic laboratory using WES, although
limited to the last two years, allowed characterization of 94 pathogenic
variants in 85 positive cases, 48 of which were novel, contributing
information to the community of disease and variant databases.