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对肌张力障碍患者进行基因检测的重要性及转化研究。

The importance of genetic testing for dystonia patients and translational research.

机构信息

Institute of Neurogenetics, University of Lübeck, Ratzeburger Allee 160, BMF, Building 67, 23538, Lübeck, Germany.

CENTOGENE GmbH, Rostock, Germany.

出版信息

J Neural Transm (Vienna). 2021 Apr;128(4):473-481. doi: 10.1007/s00702-021-02329-9. Epub 2021 Apr 19.

Abstract

Genetic testing through a variety of methods is a fundamental but underutilized approach for establishing the precise genetic diagnosis in patients with heritable forms of dystonia. Our knowledge of numerous dystonia-related genes, variants that they may contain, associated clinical presentations, and molecular disease mechanism may have significant translational potential for patients with genetically confirmed dystonia or their family members. Importantly, genetic testing permits the assembly of patient cohorts pertinent for dystonia-related research and developing therapeutics. Here we review the genetic testing approaches relevant to dystonia patients, and summarize and illustrate the multifold benefits of establishing an accurate molecular diagnosis for patients imminently or for translational research in the long run.

摘要

通过多种方法进行基因检测是确定遗传性肌张力障碍患者精确遗传诊断的基本但未充分利用的方法。我们对许多与肌张力障碍相关的基因、它们可能包含的变异、相关临床表现以及分子疾病机制的了解,对于经基因证实的肌张力障碍患者或其家庭成员可能具有重要的转化潜力。重要的是,基因检测允许为与肌张力障碍相关的研究和开发治疗方法而汇集相关患者队列。在这里,我们回顾了与肌张力障碍患者相关的基因检测方法,并总结和说明了为患者立即建立准确的分子诊断或从长远来看为转化研究建立准确的分子诊断的多重好处。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/6d14/8099821/4bc42af00652/702_2021_2329_Fig1_HTML.jpg

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