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Laboratory medicine. 2025 Apr 17:lmae127. doi: 10.1093/labmed/lmae127

Optimized efficient screening for Duchenne muscular dystrophy carriers using proto-oncogene tyrosine-protein kinase receptor Ret

优化的RET基因筛选方法在抗凝血状态下的杜氏肌营养不良症携带者筛查中的应用研究 翻译改进

Dongyang Hong  1, Zhilei Zhang  1, Yun Sun  1, Xin Wang  1, Peiying Yang  1, Tao Jiang  1, Bin Yu  2

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作者单位

  • 1 Department of Genetic Medicine Center, Women's Hospital of Nanjing Medical University (Nanjing Women and Children's Healthcare Hospital), Nanjing, China.
  • 2 Department of Medical Genetics, Changzhou Maternal and Child Health Care Hospital, Changzhou Medical Center of Nanjing Medical University, Changzhou, China.
  • DOI: 10.1093/labmed/lmae127 PMID: 40244132

    摘要 中英对照阅读

    Introduction: Duchenne muscular dystrophy (DMD) is a severe genetic disorder affecting 5% to 19% of carriers. Creatine kinase (CK) is a traditional biomarker for DMD, but its screening accuracy is limited. This study evaluated the potential of combining the proto-oncogene tyrosine-protein kinase receptor Ret (RET) with CK-MM to enhance screening efficacy.

    Methods: Creatine kinase-MM and RET levels were analyzed in 14 adult and 5 newborn carriers of DMD, along with noncarrier control individuals. The CK-MM/RET ratio was calculated, and a receiver operating characteristic curve analysis evaluated biomarker screening efficiency. Methods for extracting RET from dried blood spots (DBSs) were compared with correlations between DBSs and serum RET levels and stability under varying storage conditions.

    Results: Carriers of DMD exhibited elevated CK-MM and CK-MM/RET ratios with reduced RET. The CK-MM/RET ratio had the highest screening efficiency. Extraction of RET was optimal using Diluent C at 4 °C overnight, showing a strong DBS-serum correlation; RET remained stable, except under high humidity and temperature conditions.

    Discussion: Combining RET with CK-MM enhances DMD carrier screening, offering a more efficient DBS-based method for early detection.

    Keywords: Duchenne muscular dystrophy; RET; carrier; creatine kinase-MM; dried blood spot; proto-oncogene tyrosine-protein kinase receptor Ret.

    Keywords:efficient screening

    简介: 杜氏肌营养不良(DMD)是一种严重的遗传性疾病,影响5%到19%的携带者。肌酸激酶(CK)是用于诊断DMD的传统生物标志物,但其筛查准确性有限。本研究评估了将原癌基因酪氨酸蛋白激酶受体Ret(RET)与CK-MM结合使用以提高筛查效率的潜力。

    方法: 分析了14名成年和5名新生儿DMD携带者以及非携带对照个体中的CK-MM和RET水平。计算了CK-MM/RET比值,并通过接收器工作特性曲线分析评估了生物标志物筛查效率。比较了从干血斑(DBS)中提取RET的方法,同时考察了DBS与血液RET水平的相关性及在不同储存条件下的稳定性。

    结果: DMD携带者表现出CK-MM和CK-MM/RET比值升高而RET降低的现象。CK-MM/RET比值具有最高的筛查效率。在4°C过夜条件下使用稀释剂C提取RET效果最佳,显示出强烈的DBS-血液相关性;除了高湿度和高温条件外,RET保持稳定。

    讨论: 将RET与CK-MM结合可以增强DMD携带者的筛查,提供了一种更有效的基于干血斑的早期检测方法。

    关键词: 杜氏肌营养不良;RET;携带者;肌酸激酶-MM;干血斑;原癌基因酪氨酸蛋白激酶受体Ret。

    关键词:高效筛选; 杜氏肌营养不良携带者

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