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遗传学在遗传性出血性疾病中的应用。

The application of genetics to inherited bleeding disorders.

机构信息

Department of Medicine, Queen's University, Kingston, ON, Canada.

出版信息

Haemophilia. 2010 Jul;16 Suppl 5:35-9. doi: 10.1111/j.1365-2516.2010.02291.x.

Abstract

Most bleeding disorders encountered in clinical practice will be diagnosed, at least initially, by phenotypic assays. However, since the characterization of the genes that encode coagulation factors in the 1980s, significant progress has been made in translating this knowledge for diagnostic and therapeutic purposes. For the haemophilias, in particular, molecular genetic testing to determine carrier status, prenatal diagnosis and prediction of the likelihood of inhibitor development has now become an established component of comprehensive clinical management. For von Willebrand's disease (VWD), significant recent advances have allowed for the establishment of genotype-phenotype correlations that have improved our understanding of the disease. The availability of high density single nucleotide polymorphism (SNP) maps will allow investigators to probe the genetic basis of the general symptoms of bleeding and bruising using a comprehensive genome-wide approach. This article will review the state-of-the-art for molecular diagnostics for both haemophilia and VWD and will end with a discussion of plans for an international genome-wide association study (GWAS) designed to improve our understanding of blood coagulation.

摘要

在临床实践中遇到的大多数出血性疾病将至少在最初通过表型检测进行诊断。然而,自 20 世纪 80 年代凝血因子编码基因的特征描述以来,在将这一知识转化为诊断和治疗目的方面已经取得了重大进展。对于血友病而言,特别是用于确定携带者状态、产前诊断和预测抑制剂发展可能性的分子遗传检测,现已成为综合临床管理的既定组成部分。对于血管性血友病(VWD),最近的重大进展使得能够建立基因型-表型相关性,从而提高了我们对该疾病的认识。高密度单核苷酸多态性(SNP)图谱的可用性将使研究人员能够使用全面的全基因组方法来探究出血和瘀伤的一般症状的遗传基础。本文将回顾血友病和 VWD 的分子诊断的最新技术,并以讨论旨在提高我们对血液凝固理解的国际全基因组关联研究(GWAS)计划结束。

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