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
{"title":"【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)】。","authors":"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","doi":"10.3760/cma.j.cn511374-20250322-00173","DOIUrl":null,"url":null,"abstract":"<p><p>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.</p>","PeriodicalId":39319,"journal":{"name":"中华医学遗传学杂志","volume":"42 4","pages":"385-396"},"PeriodicalIF":0.0000,"publicationDate":"2025-04-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"[Expert consensus on the clinical application of Single-Molecule Real-Time Sequencing in the precise prevention and control of Thalassemia (2025 Edition)].\",\"authors\":\"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\",\"doi\":\"10.3760/cma.j.cn511374-20250322-00173\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>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.</p>\",\"PeriodicalId\":39319,\"journal\":{\"name\":\"中华医学遗传学杂志\",\"volume\":\"42 4\",\"pages\":\"385-396\"},\"PeriodicalIF\":0.0000,\"publicationDate\":\"2025-04-10\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"中华医学遗传学杂志\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.3760/cma.j.cn511374-20250322-00173\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q4\",\"JCRName\":\"Medicine\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"中华医学遗传学杂志","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.3760/cma.j.cn511374-20250322-00173","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q4","JCRName":"Medicine","Score":null,"Total":0}
[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.
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.