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Laminopathies: The molecular background of the disease and the prospects for its treatment

机译:拉米诺病:该疾病的分子背景及其治疗前景

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摘要

Laminopathies are rare human degenerative disorders with a wide spectrum of clinical phenotypes, associated with defects in the main protein components of the nuclear envelope, mostly in the lamins. They include systemic disorders and tissue-restricted diseases. Scientists have been trying to explain the pathogenesis of laminopathies and find an efficient method for treatment for many years. In this review, we discuss the current state of knowledge about laminopathies, the molecular mechanisms behind the development of particular phenotypes, and the prospects for stem cell and/or gene therapy treatments.
机译:椎间盘突出症是罕见的人类退行性疾病,具有广泛的临床表型,与核被膜主要蛋白成分(主要是lamin)的缺陷有关。它们包括全身性疾病和组织受限疾病。多年来,科学家一直在试图解释椎间盘突出症的发病机理,并找到一种有效的治疗方法。在这篇综述中,我们讨论了有关拉丁病的最新知识,特定表型发展的分子机制以及干细胞和/或基因疗法的前景。

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