【单分子实时测序在地中海贫血精准防控中的临床应用专家共识(2025版)】。

Q4 Medicine
Consortium For The Application Of Single-Molecule Real-Time Sequencing For The Precision Medicine And Control Of Thalassemia, Group Of Clinical Genetics Medical Genetics Branch Of Chinese Medical Doctor Association, Lingqian Wu
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引用次数: 0

摘要

地中海贫血是一种非常普遍的遗传性血红蛋白病,全世界约有3.5亿人携带红蛋白基因变体。中国南方的带菌率高达10% ~ 25%。利用血液学筛查、热点突变检测和其他技术基础上的基因检测对罕见类型的地中海贫血进行诊断的“多层次序列筛查诊断”方法,已被证明是预防和控制地中海贫血的高效方法。然而,其繁琐的流程、高昂的检测和医疗人力成本以及对遗传咨询的高需求都有明显的局限性。单分子实时测序(Single-Molecule Real-Time, SMRT)技术具有长reads和准确识别序列变异的优势,不仅拓宽了珠蛋白基因突变检测的范围,而且可以直接区分α-和β-珠蛋白基因的单倍型和结构重排,显著提高了检漏率,减少了假阳性和漏检,为地中海贫血基因变异的检测带来了革命性的变化。随着技术的成熟和成本的降低,SMRT测序在地中海贫血防控中的应用日益广泛。目前已累计临床研究50余项,临床应用数据30万条。基于这些循证研究,这一共识探讨了SMRT测序在地中海贫血临床基因检测中的应用范围、工作流程和局限性。它旨在为临床医生、实验室工作人员和决策者提供建议和指导,以支持在整个生命周期内精确预防和控制地中海贫血。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
[Expert consensus on the clinical application of Single-Molecule Real-Time Sequencing in the precise prevention and control of Thalassemia (2025 Edition)].

Thalassemia is a highly prevalent genetic hemoglobinopathy, with approximately 350 million people worldwide carrying variants of the globin genes. The carrier rate in southern China is as high as 10% ~ 25%. The "multi-level sequential screening and diagnosis" approach, which uses hematological screening, hotspot mutation testing, and genetic testing based on other technologies to diagnose rare types of thalassemia, has proven to be highly effective for the prevention and control of thalassemia. However, its cumbersome process, high cost for testing and medical labor, and high demand for genetic consulting have obvious limitations. For its advantages of long reads and accurate identification of sequence variants, Single-Molecule Real-Time (SMRT) Sequencing technology can not only broaden the scope of globin gene mutation detection, but also directly distinguish the haplotypes and structural rearrangements of the α- and β-globin genes, significantly improving the detection rate, reducing false positives and missed detection, and has revolutionized the detection for genetic variants underlying thalassemia. With the maturity of technology and decrease in cost, the application of SMRT sequencing in the prevention and control of thalassemia is becoming increasingly widespread. So far more than 50 clinical studies and 300,000 clinical application data have been accumulated. Based on these evidence-based studies, this consensus has explored the application scope, workflow, and limitations of employing SMRT sequencing in clinical genetic testing for thalassemia. It aims to provide recommendations and guidance for clinicians, laboratory staff, and policy makers, to support precise prevention and control of thalassemia throughout the full life cycle.

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来源期刊
中华医学遗传学杂志
中华医学遗传学杂志 Medicine-Medicine (all)
CiteScore
0.50
自引率
0.00%
发文量
9521
期刊介绍: Chinese Journal of Medical Genetics is a medical journal, founded in 1984, under the supervision of the China Association for Science and Technology, sponsored by the Chinese Medical Association (hosted by Sichuan University), and is now a monthly magazine, which attaches importance to academic orientation, adheres to the scientific, scholarly, advanced, and innovative, and has a certain degree of influence in the industry. Chinese Journal of Medical Genetics is a journal of Peking University, and is now included in Peking University Journal (Chinese Journal of Humanities and Social Sciences), CSCD Source Journals of Chinese Science Citation Database (with extended version), Statistical Source Journals (China Science and Technology Dissertation Outstanding Journals), Zhi.com (in Chinese), Wipu (in Chinese), Wanfang (in Chinese), CA Chemical Abstracts (U.S.), JST (Japan Science and Technology Science and Technology), and JST (Japan Science and Technology Science and Technology Research Center). ), JST (Japan Science and Technology Agency), Pж (AJ) Abstracts Journal (Russia), Copernicus Index (Poland), Cambridge Scientific Abstracts, Abstracts and Citation Database, Abstracts Magazine, Medical Abstracts, and so on.
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