[他莫昔芬，治疗所有中心核肌病的高潜力分子]。

IF 0.6 4区医学 Q4 MEDICINE, RESEARCH & EXPERIMENTAL

M S-medecine Sciences Pub Date : 2024-11-01 Epub Date: 2024-11-18 DOI:10.1051/medsci/2024130

Charlotte Gineste, Jocelyn Laporte

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引用次数: 0

摘要

中心核肌病是一种罕见的先天性疾病，以肌肉无力和细胞器错位为特征。与这些肌肉疾病相关的主要基因是 MTM1、DNM2、BIN1 和 RYR1。迄今为止，尚无治疗方法。不过，已在临床上用于治疗乳腺癌的药理化合物他莫昔芬却对中心核肌病小鼠模型的肌肉表型产生了有益的影响。在这里，我们将比较他莫昔芬对不同形式肌肉疾病的肌肉表型的影响。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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[Tamoxifen, a high-potential molecule to treat all centronuclear myopathies].

Centronuclear myopathies are rare congenital disorders characterized by muscle weakness and mislocalization of organelles. The main genes associated to these muscle diseases are MTM1, DNM2, BIN1 and RYR1. To date, no therapy is available. Nevertheless, tamoxifen, a pharmacological compound already used in clinics for breast cancer, showed beneficial effects on the muscle phenotypes in mouse models for centronuclear myopathies. Here, the effects of tamoxifen on muscle phenotypes will be compared in the various forms of this muscle disease.

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来源期刊

M S-medecine Sciences 医学-医学：研究与实验

CiteScore

0.80

自引率

14.30%

发文量

182

审稿时长

4-8 weeks

期刊介绍： m/s offers high-quality review articles in French, covering all areas of biomedical and health research, in a monthly magazine format (10 issues / year). m/s is read by the whole French-speaking community, in France but also in Belgium, Switzerland, Canada, Morocco, Algeria, Tunisia etc. m/s is not a primary publication, and thus will not consider unpublished data. Most articles are invited by the Editors, but spontaneous proposals are welcomed. Each issue combines news and views on the most recent scientific publications, as well as broadly accessible and updated review articles on a specific topic, and essays on science and society, history of science, public health, or reactions to published articles. Each year, m/s also publishes one or two thematic issues focused on a research topic of high interest. All review articles and essays are peer-reviewed.