Comprehensive whole genome sequence analyses yields novel genetic and structural insights for Intellectual Disability

<h3>Background</h3><p dir="ltr">Intellectual Disability (ID) is among the most common global disorders, yet etiology is unknown in ~30% of patients despite clinical assessment. Whole genome sequencing (WGS) is able to interrogate the entire genome, providing potential to...

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Main Author: Farah R. Zahir (18892108) (author)
Other Authors: Jill C. Mwenifumbo (19725358) (author), Hye-Jung E. Chun (19725361) (author), Emilia L. Lim (3348266) (author), Clara D. M. Van Karnebeek (19725364) (author), Madeline Couse (3385817) (author), Karen L. Mungall (9709370) (author), Leora Lee (3214851) (author), Nancy Makela (4046018) (author), Linlea Armstrong (4046036) (author), Cornelius F. Boerkoel (12242496) (author), Sylvie L. Langlois (19725367) (author), Barbara M. McGillivray (19725370) (author), Steven J. M. Jones (9709382) (author), Jan M. Friedman (11252261) (author), Marco A. Marra (7610399) (author)
Published: 2017
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Summary:<h3>Background</h3><p dir="ltr">Intellectual Disability (ID) is among the most common global disorders, yet etiology is unknown in ~30% of patients despite clinical assessment. Whole genome sequencing (WGS) is able to interrogate the entire genome, providing potential to diagnose idiopathic patients.</p><h3>Methods</h3><p dir="ltr">We conducted WGS on eight children with idiopathic ID and brain structural defects, and their normal parents; carrying out an extensive data analyses, using standard and discovery approaches.</p><h3>Results</h3><p dir="ltr">We verified <i>de novo</i> pathogenic single nucleotide variants (SNV) in <i>ARID1B c.1595delG</i> and <i>PHF6 c.820C > T</i>, potentially causative <i>de novo</i> two base indels in <i>SQSTM1 c.115_116delinsTA</i> and <i>UPF1 c.1576_1577delinsA,</i> and <i>de novo</i> SNVs in <i>CACNB3 c.1289G > A,</i> and <i>SPRY4 c.508 T > A,</i> of uncertain significance. We report results from a large secondary control study of 2081 exomes probing the pathogenicity of the above genes. We analyzed structural variation by four different algorithms including <i>de novo</i> genome assembly. We confirmed a likely contributory 165 kb <i>de novo</i> heterozygous 1q43 microdeletion missed by clinical microarray. The <i>de novo</i> assembly resulted in unmasking hidden genome instability that was missed by standard re-alignment based algorithms. We also interrogated regulatory sequence variation for known and hypothesized ID genes and present useful strategies for WGS data analyses for non-coding variation.</p><h3>Conclusion</h3><p dir="ltr">This study provides an extensive analysis of WGS in the context of ID, providing genetic and structural insights into ID and yielding diagnoses.</p><h2>Other Information</h2><p dir="ltr">Published in: BMC Genomics<br>License: <a href="https://creativecommons.org/licenses/by/4.0/deed.en" rel="noreferrer noopener" target="_blank">https://creativecommons.org/licenses/by/4.0/</a>  <br>See article on publisher's website: <a href="https://dx.doi.org/10.1186/s12864-017-3671-0" target="_blank">https://dx.doi.org/10.1186/s12864-017-3671-0</a></p>