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遗传性出血性、血栓性和血小板疾病基因检测解读的改善。

Improving interpretation of genetic testing for hereditary hemorrhagic, thrombotic, and platelet disorders.

机构信息

Division of Hematology, The Children's Hospital of Philadelphia, Philadelphia, PA; and Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA.

出版信息

Hematology Am Soc Hematol Educ Program. 2020 Dec 4;2020(1):76-81. doi: 10.1182/hematology.2020000091.

Abstract

The last 10 years have seen an explosion in the amount of data available through next-generation sequencing. These data are advancing quickly, and this pace makes it difficult for most practitioners to easily keep up with all of the new information. Complicating this understanding is sometimes conflicting information about variant pathogenicity or even about the role of some genes in the pathogenesis of disease. The more widespread clinical use of sequencing has expanded phenotypes, including the identification of mild phenotypes associated with previously serious disease, such as with some variants in RUNX1, MYH9, ITG2A, and others. Several organizations have taken up the task of cataloging and systematically evaluating genes and variants using a standardized approach and making the data publicly available so that others can benefit from their gene/variant curation. The efforts in testing for hereditary hemorrhagic, thrombotic, and platelet disorders have been led by the International Society on Thrombosis and Haemostasis Scientific Standardization Committee on Genomics in Thrombosis and Hemostasis, the American Society of Hematology, and the National Institutes of Health National Human Genome Research Institute Clinical Genome Resource. This article outlines current efforts to improve the interpretation of genetic testing and the role of standardizing and disseminating information. By assessing the strength of gene-disease associations, standardizing variant curation guidelines, sharing genomic data among expert members, and incorporating data from existing disease databases, the number of variants of uncertain significance will decrease, thereby improving the value of genetic testing as a diagnostic tool.

摘要

过去 10 年中,通过下一代测序获得的数据呈爆炸式增长。这些数据发展迅速,其更新速度之快使得大多数从业者难以轻松掌握所有新信息。更复杂的是,有关变异致病性的信息有时会相互矛盾,甚至有些基因在疾病发病机制中的作用也存在争议。测序在临床上的广泛应用扩大了表型,包括发现以前严重疾病相关的轻度表型,如 RUNX1、MYH9、ITG2A 等基因的某些变体。为了系统地评估基因和变体,几个组织采用标准化方法对其进行分类,并公开数据,以便其他人能够从基因/变体的管理中受益。遗传性出血性、血栓性和血小板疾病检测方面的工作由国际血栓形成与止血学会基因组学血栓形成与止血科学标准化委员会、美国血液学会和美国国立卫生研究院国家人类基因组研究所临床基因组资源牵头。本文概述了当前改善遗传检测解释的努力以及标准化和传播信息的作用。通过评估基因-疾病关联的强度、标准化变体管理指南、在专家成员之间共享基因组数据以及整合来自现有疾病数据库的数据,将减少不确定意义的变异数量,从而提高遗传检测作为诊断工具的价值。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/41d7/7727548/421d4e01ea50/bloodbook-2020-76-absf1.jpg

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