Open Access

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

  • Magdalena Zaremba-Czogalla1,
  • Magda Dubińska-Magiera1 and
  • Ryszard Rzepecki1Email author
Cellular & Molecular Biology LettersAn International Journal201016:38

DOI: 10.2478/s11658-010-0038-9

Received: 28 September 2010

Accepted: 13 December 2010

Published: 27 December 2010

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.

Key words

Laminopathies Nuclear lamina Lamin Emerin Gene therapy

Advertisement