Language

English

Publication Date

12-1-2022

Journal

Human Mutation

DOI

10.1002/humu.24497

PMID

36317458

PMCID

PMC9772143

PubMedCentral® Posted Date

12-1-2023

PubMedCentral® Full Text Version

Author MSS

Abstract

Advanced bioinformatics algorithms allow detection of multiple-exon copy-number variations (CNVs) from exome sequencing (ES) data, while detection of single-exon CNVs remains challenging. A retrospective review of Baylor Genetics' clinical ES patient cohort identified four individuals with homozygous single-exon deletions of TBCK (exon 23, NM_001163435.2), a gene associated with an autosomal recessive neurodevelopmental phenotype. To evaluate the prevalence of this deletion and its contribution to disease, we retrospectively analyzed single nucleotide polymorphism (SNP) array data for 8194 individuals undergoing ES, followed by PCR confirmation and RT-PCR on individuals carrying homozygous or heterozygous exon 23 TBCK deletions. A fifth individual was diagnosed with the TBCK-related disorder due to a heterozygous exon 23 deletion in trans with a c.1860+1G>A (NM_001163435.2) pathogenic variant, and three additional heterozygous carriers were identified. Affected individuals and carriers were from diverse ethnicities including European Caucasian, South Asian, Middle Eastern, Hispanic American and African American, with only one family reporting consanguinity. RT-PCR revealed two out-of-frame transcripts related to the exon 23 deletion. Our results highlight the importance of identifying single-exon deletions in clinical ES, especially for genes carrying recurrent deletions. For patients with early-onset hypotonia and psychomotor delay, this single-exon TBCK deletion might be under-recognized due to technical limitations of ES.

Keywords

Humans, DNA Copy Number Variations, Exome, Exome Sequencing, Exons, Muscle Hypotonia, Muscular Diseases, Protein Serine-Threonine Kinases, Retrospective Studies, Infant, TBCK, single-exon deletion, exome sequencing, diagnostic testing, recurrent variant

Published Open-Access

yes

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