The neuronal ceroid lipofuscinoses: Opportunities from model systems

Research output: Contribution to journalArticle

Authors

  • Kiterie M E Faller
  • Rodrigo Gutierrez-Quintana
  • Alamin Mohammed
  • Ahad A Rahim
  • Kim Wager
  • Michael Bond

External organisations

  • School of Veterinary Medicine, College of Veterinary, Medical and Life Sciences, Bearsden Road, Glasgow G61 1QH, UK.
  • College of Medical and Dental Sciences; University of Birmingham; Birmingham B15 2TT UK
  • UCL School of Pharmacy, 29-39 Brunswick Square, London WC1N 1AX, UK.
  • Cardiff University
  • University College London

Abstract

The neuronal ceroid lipofuscinoses are a group of severe and progressive neurodegenerative disorders, generally with childhood onset. Despite the fact that these diseases remain fatal, significant breakthroughs have been made in our understanding of the genetics that underpin these conditions. This understanding has allowed the development of a broad range of models to study disease processes, and to develop new therapeutic approaches. Such models have contributed significantly to our knowledge of these conditions. In this review we will focus on the advantages of each individual model, describe some of the contributions the models have made to our understanding of the broader disease biology and highlight new techniques and approaches relevant to the study and potential treatment of the neuronal ceroid lipofuscinoses. This article is part of a Special Issue entitled: "Current Research on the Neuronal Ceroid Lipofuscinoses (Batten Disease)".

Details

Original languageEnglish
Pages (from-to)2267-78
Number of pages12
JournalBiochimica et Biophysica Acta
Volume1852
Issue number10 Pt B
Publication statusPublished - Oct 2015

Keywords

  • Journal Article, Review