Li Weihao, Ye Yanchou
Reproductive Medicine Center, The Seventh Affiliated Hospital of Sun Yat-sen University, Shenzhen, China.
Prenatal Diagnostic Center, Medical Genetics Center, Department of Obstetrics and Gynaecology, The Seventh Affiliated Hospital of Sun Yat-sen University, 628 Zhenyuan Rd., Guangming Dist., Shenzhen, China.
Mol Cytogenet. 2024 Dec 18;17(1):32. doi: 10.1186/s13039-024-00701-4.
Thalassemia is an autosomal recessive genetic disorder and a common form of Hemoglobinopathy. It is classified into α-thalassemia and β-thalassemia. This disease is mainly prevalent in tropical and subtropical regions, including southern China. Severe α-thalassemia and intermediate α-thalassemia are among the most common birth defects in southern China. Intermediate α-thalassemia, also known as Hb H disease, is characterized by moderate anemia. Severe α-thalassemia, also known as Hb Bart's Hydrops fetalis syndrome, is a fatal condition. Infants with severe β-thalassemia do not show symptoms at birth but develop severe anemia later, requiring expensive treatment. Most untreated patients with severe β-thalassemia die in early childhood. Screening for thalassemia carriers and genetic diagnosis in high-prevalence areas significantly reduce the incidence of severe thalassemia. This review aims to summarize the genetic diagnostic approaches for thalassemia. Conventional genetic testing methods can identify 95-98% of thalassemia carriers but may miss rare thalassemia genotypes. Third-Generation Sequencing offers significant advantages in complementing other genetic diagnostic approaches, providing a basis for genetic counseling and prenatal diagnosis.
地中海贫血是一种常染色体隐性遗传疾病,也是血红蛋白病的常见形式。它分为α地中海贫血和β地中海贫血。这种疾病主要流行于热带和亚热带地区,包括中国南方。重型α地中海贫血和中间型α地中海贫血是中国南方最常见的出生缺陷之一。中间型α地中海贫血,也称为血红蛋白H病,其特征为中度贫血。重型α地中海贫血,也称为巴氏水肿胎儿综合征,是一种致命疾病。重型β地中海贫血婴儿出生时无症状,但后期会发展为严重贫血,需要昂贵的治疗。大多数未经治疗的重型β地中海贫血患者在幼儿期死亡。在高发地区对地中海贫血携带者进行筛查和基因诊断可显著降低重型地中海贫血的发病率。本综述旨在总结地中海贫血的基因诊断方法。传统基因检测方法可识别95%至98%的地中海贫血携带者,但可能遗漏罕见的地中海贫血基因型。第三代测序在补充其他基因诊断方法方面具有显著优势,为遗传咨询和产前诊断提供了依据。