Unified long-read panel for Parkinson’s and repeat expansion disorders

Abstract In this study, we designed a gene panel based on Nanopore long-read sequencing using adaptive sampling, targeting n = 564 genes associated with Parkinson’s disease (PD) and repeat expansion disorders. We investigated its diagnostic utility in n = 18 patients with (1) pathogenic variants in LRRK2 , PRKN , SNCA , and RAB32 ( n = 7); (2) idiopathic PD or FTD negative for known genetic causes ( n = 3); (3) repeat expansions in ATXN1 , ATXN2 , ATXN3 , C9orf72 , DAB1 , FGF14 , HTT , and TAF1 -SVA ( n = 8). Three individuals were multiplexed per flow cell, achieving a mean coverage of 24X (SD = ± 9X) per sample. Ultimately, 17/20 (85%; Clopper–Pearson 95% CI: 62–97%) expected pathogenic variants were identified; i.e., an SNCA triplication and two repeat expansions in C9orf72 and TAF1 -SVA were missed. Additional variants in STXBP2 , AP4S1 , RARS2 , ALS2 , and CNBP were identified in five patients. Adaptive sampling is a versatile genetic diagnostic tool in neurodegenerative disorders, while enabling cost-effective multiplexing. Further validation and improvements in bioinformatic analysis are needed.

Authors

Publication Details

Journal
npj Parkinson s Disease
Published
2026-09-24
DOI
https://doi.org/10.1038/s41531-026-01585-4
Primary Topic
Parkinson's Disease Mechanisms and Treatments
Type
article
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article

Unified long-read panel for Parkinson’s and repeat expansion disorders

A. Nazlı Başak, Roland Dominic G. Jamora, Gerard Saranza, Theresa Lüth et al.
npj Parkinson s Disease
Parkinson's Disease Mechanisms and Treatments
article

Unified long-read panel for Parkinson’s and repeat expansion disorders

A. Nazlı Başak, Roland Dominic G. Jamora, Gerard Saranza, Theresa Lüth, Carolin Gabbert, Elisabeth Stögmann, Alexander Balck, Cid Czarina E. Diesta, Susen Schaake, Christoph Much, Philip Seibler, Teresa Kleinz, Max Borsche, Joshua Laβ, Tuğçe Gül, Norbert Brüggemann, Theresa König, Raymond L. Rosales, André Fienemann, Julia C. Prietzsche, Meret Möller, Alexander Zimprich, Christine Klein, Joanne Trinh, Christos Ganos
article en

Abstract

Abstract In this study, we designed a gene panel based on Nanopore long-read sequencing using adaptive sampling, targeting n = 564 genes associated with Parkinson’s disease (PD) and repeat expansion disorders. We investigated its diagnostic utility in n = 18 patients with (1) pathogenic variants in LRRK2 , PRKN , SNCA , and RAB32 ( n = 7); (2) idiopathic PD or FTD negative for known genetic causes ( n = 3); (3) repeat expansions in ATXN1 , ATXN2 , ATXN3 , C9orf72 , DAB1 , FGF14 , HTT , and TAF1 -SVA ( n = 8). Three individuals were multiplexed per flow cell, achieving a mean coverage of 24X (SD = ± 9X) per sample. Ultimately, 17/20 (85%; Clopper–Pearson 95% CI: 62–97%) expected pathogenic variants were identified; i.e., an SNCA triplication and two repeat expansions in C9orf72 and TAF1 -SVA were missed. Additional variants in STXBP2 , AP4S1 , RARS2 , ALS2 , and CNBP were identified in five patients. Adaptive sampling is a versatile genetic diagnostic tool in neurodegenerative disorders, while enabling cost-effective multiplexing. Further validation and improvements in bioinformatic analysis are needed.

npj Parkinson s DiseaseVol. 12(1)
Openalex Percentile: Top 12%
Parkinson's Disease Mechanisms and Treatments
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