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镶嵌条件下的意图测试:基于案例的综述。

Testing With Intent in Mosaic Conditions: A Case-Based Review.

作者信息

Kerwin Andrew J, Lop Ana L, Vicente Kristyn, Weiler Tracey, Kana Sajel L

机构信息

Department of Genetics, Florida International University, Herbert Wertheim College of Medicine, Miami, USA.

Department of Genetics, New York Medical College, Valhalla, USA.

出版信息

Cureus. 2023 Nov 29;15(11):e49644. doi: 10.7759/cureus.49644. eCollection 2023 Nov.

Abstract

Recent advancements in genetic testing have revealed cases of mosaicism, demonstrating the phenomenon may be more common than once thought. Broadly defined, mosaicism describes the presence of two genotypically different cell lineages within the same organism. This can arise from small mutations or errors in chromosome segregation, as early as in gametes, before or after fertilization. Mosaicism is directly responsible for many conditions that present in a wide range of tissues, with the presence of the mutation or genetic abnormality following a tissue-dependent pattern. This makes it possible for patients to test negative for a condition using a standard tissue sample while harboring the variant in a different tissue. Understanding the timing and mechanisms of mosaic conditions will aid in targeted testing that is more appropriate to identify a pathogenic variant. This targeted testing should reduce the length of a patient's diagnostic odyssey and provide a better understanding of the chances of passing on their variant to their offspring, thereby allowing for more accurate genetic counseling. We illustrate this phenomenon with two cases: one of Pallister-Killian syndrome and the other of tuberous sclerosis complex. Both patients had increased time to diagnosis because of difficulties in identifying genetic variants in tested tissues. Beyond just increased time to diagnosis, we illustrate that mosaic conditions can present as less severe and more variable than the germline condition and how specific germ layers may be affected by the variant. Knowing which germ layers may be affected by the variant can give clinicians a clue as to which tissues may need to be tested to yield the most accurate result.

摘要

基因检测领域的最新进展揭示了嵌合体病例,表明这种现象可能比人们曾经认为的更为普遍。广义上讲,嵌合体是指同一生物体内存在两种基因型不同的细胞谱系。这可能源于染色体分离过程中的小突变或错误,早在配子形成时,即受精之前或之后就可能发生。嵌合体直接导致了许多出现在多种组织中的病症,突变或基因异常的存在遵循组织依赖模式。这使得患者在使用标准组织样本检测某种病症呈阴性时,而在其他组织中却携带该变异成为可能。了解嵌合病症的发生时间和机制将有助于进行更有针对性的检测,从而更适当地识别致病变异。这种有针对性的检测应能缩短患者的诊断历程,并更好地了解将变异遗传给后代的几率,从而实现更准确的遗传咨询。我们用两个病例来说明这种现象:一个是帕利斯特 - 基利安综合征,另一个是结节性硬化症。由于在检测组织中识别基因变异存在困难,这两名患者的诊断时间都延长了。除了诊断时间延长之外,我们还说明了嵌合病症可能表现得比种系病症症状较轻且变化更大,以及特定胚层可能如何受到变异的影响。了解哪些胚层可能受到变异影响,可以为临床医生提供线索,提示哪些组织可能需要进行检测以获得最准确的结果。

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