Unraveling MECP2 structural variants in previously elusive Rett syndrome cases through IGV interpretation.

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Tác giả: Nadav Agam, Ilana Aminov, Bruria Ben-Zeev, Ohad S Birk, John Christodoulou, Vadim Dolgin, Ofek Freund, Noam Hadar, Gali Heimer, Matan M Jean, Simran Kaur, Tomer Poleg, Amit Safran

Ngôn ngữ: eng

Ký hiệu phân loại:

Thông tin xuất bản: England : NPJ genomic medicine , 2025

Mô tả vật lý:

Bộ sưu tập: NCBI

ID: 711414

 Rett syndrome (RTT) is a severe neurodevelopmental disorder, with MECP2 mutations accounting for 90-95% of classic and 50-70% of atypical cases. However, many clinically diagnosed RTT patients remain without molecular diagnoses. While point mutations and large rearrangements in MECP2 are well studied, the role of small-intermediate structural variants (SVs) remains mostly elusive. Using standard short-read whole genome sequencing, we identified novel de novo SVs in three out of three previously unresolved RTT cases: a complex SV with two deletions ( ~ 5Kbp and ~60Kbp) and a ~105Kbp inversion
  a ~200Kbp translocation
  and a ~3Kbp deletion. These findings suggest that such elusive SVs might be a common cause for "MECP2-negative" RTT. Incorporating SV detection into routine genetic testing through bioinformatic analysis of short-read sequencing or manual review using IGV could improve diagnostic rates and expand our understanding of RTT and similar disorders.
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