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肌酸激酶-MM/原癌基因酪氨酸蛋白激酶受体作为杜氏肌营养不良症携带者的敏感指标。

Creatine Kinase-MM/Proto-oncogene Tyrosine-Protein Kinase Receptor as a Sensitive Indicator for Duchenne Muscular Dystrophy Carriers.

机构信息

Center of Genetic Medicine, The affiliated Obstetrics and Gynecology Hospital with Nanjing Medical University, Nanjing Women and Children's Healthcare Hospital, Nanjing, Jiangsu, China.

, Nanjing, China.

出版信息

Mol Neurobiol. 2024 Dec;61(12):10645-10655. doi: 10.1007/s12035-024-04235-z. Epub 2024 May 20.

Abstract

Duchenne muscular dystrophy (DMD), a lethal X-linked recessive genetic disease, is characterized by progressive muscle wasting which will lead to premature death by cardiorespiratory complications in their late twenties. And 2.5-19% DMD carriers that also suffer from skeletal muscle damage or dilated cardiomyopathy when diagnosed as soon as possible is meaningful for prenatal diagnosis and advance warning for self-health. The current DMD carrier screening mainly relies on detecting serum creatine kinase activity, covering only 50-70% DMD carriers which will cause many false negatives and require the discovery of highly effective biomarker and simple detection procedure for DMD carriers. In this article, we have compiled a comprehensive summary of all documented biomarkers associated with DMD and categorized them based on their expression patterns. We specifically pinpointed novel DMD biomarkers, previously unreported in DMD carriers, and conducted further investigations to explore their potential. Compared to creatine kinase activity alone in DMD carriers, creatine kinase-MM can improve the specificity from 73 to 81%. And our investigation revealed another promising protein: proto-oncogene tyrosine-protein kinase receptor (RET). When combined with creatine kinase-MM (creatine kinase-MM/RET ratio), it significantly enhances the specificity (from 81 to 83%) and sensitivity (from 71.4 to 93%) of detecting DMD carriers in serum. Moreover, we successfully devised an efficient method for extracting RET from dried blood spots. This breakthrough allowed us to detect both creatine kinase-MM and RET using dried blood spots without compromising the detection rate.

摘要

杜氏肌营养不良症(DMD)是一种致命的 X 连锁隐性遗传病,其特征是进行性肌肉萎缩,在二十多岁时会因心肺并发症而导致过早死亡。而 2.5-19%的 DMD 携带者在被诊断时也会出现骨骼肌损伤或扩张型心肌病,这对产前诊断和自我健康预警具有重要意义。目前的 DMD 携带者筛查主要依赖于检测血清肌酸激酶活性,仅覆盖 50-70%的 DMD 携带者,这会导致许多假阴性结果,因此需要发现高度有效的生物标志物和简单的检测程序来筛查 DMD 携带者。在本文中,我们汇总了所有与 DMD 相关的已记录生物标志物,并根据其表达模式进行了分类。我们特别指出了以前在 DMD 携带者中未报告过的新型 DMD 生物标志物,并进行了进一步的研究来探索它们的潜力。与单独检测肌酸激酶活性相比,肌酸激酶-MM 可将特异性从 73%提高到 81%。我们的研究还揭示了另一种有前途的蛋白质:原癌基因酪氨酸蛋白激酶受体(RET)。当与肌酸激酶-MM (肌酸激酶-MM/RET 比值)结合使用时,它可显著提高血清中 DMD 携带者的特异性(从 81%提高到 83%)和敏感性(从 71.4%提高到 93%)。此外,我们成功设计了一种从干血斑中提取 RET 的有效方法。这一突破使我们能够在不降低检测率的情况下使用干血斑同时检测肌酸激酶-MM 和 RET。

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