High-resolution repeat structure analysis in myotonic dystrophy type 2 diagnostics using short-read whole genome sequencing.

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Tác giả: Andrej Balaz, Jaroslav Budis, Ludevit Kadasi, Marcel Kucharik, Ingrid Lojova, Zuzana Pös, Jan Radvanszky, Tomas Szemes, Eva Tothova Tarova, Andrea Zatkova

Ngôn ngữ: eng

Ký hiệu phân loại: 230.071 Education in Christianity, in Christian theology

Thông tin xuất bản: United States : Analytical biochemistry , 2025

Mô tả vật lý:

Bộ sưu tập: NCBI

ID: 97106

BACKGROUND/OBJECTIVES: Diagnostic possibilities for myotonic dystrophy type 2 (DM2) are constantly evolving in order to achieve more accurate and faster diagnosis. Whole genome sequencing (WGS), together with specialized tandem repeat (TR) genotyping bioinformatic tools, represent a breakthrough technology in molecular diagnostics. We decided to characterize new opportunities and challenges in WGS-based DM2 molecular diagnostics. METHODS: WGS data were obtained from 50 individuals, including five DM2 patients, and one individual carrying a premutation range allele. TR characterization was performed using a modified version of the Dante tool, with results validated by conventional PCR and repeat-primed PCR. RESULTS: We used WGS to identify all of the expansion-range DM2 alleles, together with the premutation-range allele. Compared to conventional methods, WGS was more efficient for a detailed sequence structure characterization of the normal-range alleles, and phasing of the entire CNBP-complex motif. A 97 % genotyping concordance rate was achieved between the conventional methods and the WGS-derived results, with discrepancies mainly based on single-repeat differences in the genotypes. The stutter effect introduced some uncertainty in both methods. CONCLUSION: Short-read WGS offers significant potential for DM2 diagnostics by enabling precise repeat motif characterization and may also apply to other tandem repeat disorders (TRDs).
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