[HTML][HTML] Laminopathies: the molecular background of the disease and the prospects for its treatment

M Zaremba-Czogalla, M Dubińska-Magiera… - Cellular and Molecular …, 2011 - degruyter.com
M Zaremba-Czogalla, M Dubińska-Magiera, R Rzepecki
Cellular and Molecular Biology Letters, 2011degruyter.com
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 …
Abstract
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.
De Gruyter