Identification of a de novo MYH9 mutation in a Chinese family with MYH9-related disease

Objectives MYH9-related disease (MYH9-RD) is a congenital bleeding disorder characterized by thrombocytopenia, platelet macrocytosis, inclusion bodies in neutrophils.The aim of this study was to investigate a Chinese family with MYH9-RD and to identity potential mutations of the MYH9. This investiga...

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Main Authors: Ruimin Cai, Shiyu Bai, Yanjun Liu, Fei Li, Wenyang Wang, Yi Li, Qiang Feng
Format: Article
Language:English
Published: Taylor & Francis Group 2025-12-01
Series:Hematology
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Online Access:https://www.tandfonline.com/doi/10.1080/16078454.2025.2532923
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author Ruimin Cai
Shiyu Bai
Yanjun Liu
Fei Li
Wenyang Wang
Yi Li
Qiang Feng
author_facet Ruimin Cai
Shiyu Bai
Yanjun Liu
Fei Li
Wenyang Wang
Yi Li
Qiang Feng
author_sort Ruimin Cai
collection DOAJ
description Objectives MYH9-related disease (MYH9-RD) is a congenital bleeding disorder characterized by thrombocytopenia, platelet macrocytosis, inclusion bodies in neutrophils.The aim of this study was to investigate a Chinese family with MYH9-RD and to identity potential mutations of the MYH9. This investigation will elucidate the molecular mechanisms involved in disease pathogenesis.Methods Whole exome sequencing (WES) followed by Sanger sequencing was conducted on all family members. Multiple bioinformatics tools were programmed to predict the conservation of mutations and the effect on the protein structure, including 3D protein model analysis.Results Gene sequencing revealed that the patient carried A de novo missense MYH9 mutation (c.4338T > G, p.Phe1446Leu). Bioinformatics and molecular modeling analyses predict the p.Phe1446Leu variant to be deleterious, with probable disruptive effects on protein structure and consequent functional impairment.Discussion Our study suggested that a de novo missense mutation in MYH9 may be causative to MYH9-RD. Furthermore, genetic sequencing is expected to soon become a standard component of the diagnostic evaluation for individuals with platelet disorders.
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spelling doaj-art-51fc392f66cb44f8980c368bcbdd208f2025-08-20T03:28:13ZengTaylor & Francis GroupHematology1607-84542025-12-0130110.1080/16078454.2025.2532923Identification of a de novo MYH9 mutation in a Chinese family with MYH9-related diseaseRuimin Cai0Shiyu Bai1Yanjun Liu2Fei Li3Wenyang Wang4Yi Li5Qiang Feng6Department of Clinical Laboratory, The Affiliated Taian City Central Hospital of Qingdao University, Taian, People’s Republic of ChinaDepartment of Clinical Laboratory, The Affiliated Taian City Central Hospital of Qingdao University, Taian, People’s Republic of ChinaDepartment of Clinical Laboratory, The Affiliated Taian City Central Hospital of Qingdao University, Taian, People’s Republic of ChinaDepartment of Clinical Laboratory, The Affiliated Taian City Central Hospital of Qingdao University, Taian, People’s Republic of ChinaDepartment of Clinical Laboratory, The Affiliated Taian City Central Hospital of Qingdao University, Taian, People’s Republic of ChinaDepartment of Clinical Laboratory, The Affiliated Taian City Central Hospital of Qingdao University, Taian, People’s Republic of ChinaDepartment of Clinical Laboratory, The Affiliated Taian City Central Hospital of Qingdao University, Taian, People’s Republic of ChinaObjectives MYH9-related disease (MYH9-RD) is a congenital bleeding disorder characterized by thrombocytopenia, platelet macrocytosis, inclusion bodies in neutrophils.The aim of this study was to investigate a Chinese family with MYH9-RD and to identity potential mutations of the MYH9. This investigation will elucidate the molecular mechanisms involved in disease pathogenesis.Methods Whole exome sequencing (WES) followed by Sanger sequencing was conducted on all family members. Multiple bioinformatics tools were programmed to predict the conservation of mutations and the effect on the protein structure, including 3D protein model analysis.Results Gene sequencing revealed that the patient carried A de novo missense MYH9 mutation (c.4338T > G, p.Phe1446Leu). Bioinformatics and molecular modeling analyses predict the p.Phe1446Leu variant to be deleterious, with probable disruptive effects on protein structure and consequent functional impairment.Discussion Our study suggested that a de novo missense mutation in MYH9 may be causative to MYH9-RD. Furthermore, genetic sequencing is expected to soon become a standard component of the diagnostic evaluation for individuals with platelet disorders.https://www.tandfonline.com/doi/10.1080/16078454.2025.2532923MYH9-related diseasemacrothrombocytopeniaDNA analysiscoiled-coil domain
spellingShingle Ruimin Cai
Shiyu Bai
Yanjun Liu
Fei Li
Wenyang Wang
Yi Li
Qiang Feng
Identification of a de novo MYH9 mutation in a Chinese family with MYH9-related disease
Hematology
MYH9-related disease
macrothrombocytopenia
DNA analysis
coiled-coil domain
title Identification of a de novo MYH9 mutation in a Chinese family with MYH9-related disease
title_full Identification of a de novo MYH9 mutation in a Chinese family with MYH9-related disease
title_fullStr Identification of a de novo MYH9 mutation in a Chinese family with MYH9-related disease
title_full_unstemmed Identification of a de novo MYH9 mutation in a Chinese family with MYH9-related disease
title_short Identification of a de novo MYH9 mutation in a Chinese family with MYH9-related disease
title_sort identification of a de novo myh9 mutation in a chinese family with myh9 related disease
topic MYH9-related disease
macrothrombocytopenia
DNA analysis
coiled-coil domain
url https://www.tandfonline.com/doi/10.1080/16078454.2025.2532923
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