title: Laminopathies: the molecular background of the disease and the prospects for its treatment.
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.
authors.affiliation: Laboratory of Nuclear Proteins, Faculty of Biotechnology, University of Wrocław, ul. Przybyszewskiego 63/77, 51-148, Wroclaw, Poland.
authors.forename: Magdalena
authors.initials: M
authors.lastname: Zaremba-Czogalla
authors.forename: Magda
authors.initials: M
authors.lastname: Dubińska-Magiera
authors.forename: Ryszard
authors.initials: R
authors.lastname: Rzepecki
mesh_terms: D030342:Genetic Diseases, Inborn / Q000235:genetics* / Q000628:therapy; D015316:Genetic Therapy; D006801:Humans; D034882:Lamins / Q000235:genetics* / Q000378:metabolism; D008060:Lipodystrophy / Q000628:therapy; D009135:Muscular Diseases / Q000628:therapy; D009685:Nuclear Envelope / Q000378:metabolism; D011371:Progeria / Q000628:therapy
chemical_list: D034882:Lamins