Unravelling undiagnosed rare disease cases by HiFi long-read genome sequencing

W Steyaert, L Sagath, G Demidov, VA Yepez… - medRxiv, 2024 - medrxiv.org
Solve-RD is a pan-European rare disease (RD) research program that aims to identify
disease-causing genetic variants in previously undiagnosed RD families. We utilized 10-fold …

[HTML][HTML] Long-read genome sequencing and variant reanalysis increase diagnostic yield in neurodevelopmental disorders

SM Hiatt, JMJ Lawlor, LH Handley, DR Latner… - medRxiv, 2024 - ncbi.nlm.nih.gov
Variant detection from long-read genome sequencing (lrGS) has proven to be considerably
more accurate and comprehensive than variant detection from short-read genome …

Solving unsolved rare neurological diseases—a Solve-RD viewpoint

R Schüle, D Timmann, CE Erasmus… - European Journal of …, 2021 - nature.com
Rare genetic neurological disorders (RND; ORPHA: 71859) are a heterogeneous group of
disorders comprising> 1700 distinct genetic disease entities. However, genetic discoveries …

Unique Capabilities of Genome Sequencing for Rare Disease Diagnosis

MH Wojcik, G Lemire, MS Zaki, M Wissman, W Win… - medRxiv, 2023 - medrxiv.org
Background Causal variants underlying rare disorders may remain elusive even after
expansive gene panels or exome sequencing (ES). Clinicians and researchers may then …

Comprehensive de novo mutation discovery with HiFi long-read sequencing

E Kucuk, BPGH van der Sanden, L O'Gorman… - Genome Medicine, 2023 - Springer
Background Long-read sequencing (LRS) techniques have been very successful in
identifying structural variants (SVs). However, the high error rate of LRS made the detection …

[HTML][HTML] Genomic answers for children: Dynamic analyses of> 1000 pediatric rare disease genomes

ASA Cohen, EG Farrow, AT Abdelmoity, JT Alaimo… - Genetics in …, 2022 - Elsevier
Purpose This study aimed to provide comprehensive diagnostic and candidate analyses in a
pediatric rare disease cohort through the Genomic Answers for Kids program. Methods …

Targeted long-read sequencing resolves complex structural variants and identifies missing disease-causing variants

DE Miller, A Sulovari, T Wang, H Loucks, K Hoekzema… - BioRxiv, 2020 - biorxiv.org
ABSTRACT BACKGROUND Despite widespread availability of clinical genetic testing, many
individuals with suspected genetic conditions do not have a precise diagnosis. This limits …

Gaps in the phenotype descriptions of ultra-rare genetic conditions: review and multi-center consensus reporting guidelines

A AlMail, A Jamjoom, A Pan, MY Feng, V Chau… - medRxiv, 2023 - medrxiv.org
Background: Genome-wide sequencing and genetic matchmaker services are propelling a
new era of genotype-first ascertainment of novel genetic conditions. The degree to which …

Deciphering the role of germline complex de novo structural variations in rare disorders

H Jung, TP Yang, S Walker, P Danecek, IG Salinas… - bioRxiv, 2024 - biorxiv.org
De novo structural variants (dnSVs) have emerged as crucial genetic factors in the context of
rare disorders. However, these variations often go undiagnosed in routine genetic screening …

Editorial–Diagnostic genome sequencing in rare disorders

P Krawitz, TB Haack - Medizinische Genetik, 2023 - degruyter.com
About 8,000 rare diseases (RDs) are currently known. Although rare by definition
(prevalence of 1: 2,000 in the European population), they collectively constitute a major …