The neuronal ceroid lipofuscinoses: Opportunities from model systems.
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)".
School of Veterinary Medicine, College of Veterinary, Medical and Life Sciences, Bearsden Road, Glasgow G61 1QH, UK.
Kiterie M E
KM
Faller
School of Veterinary Medicine, College of Veterinary, Medical and Life Sciences, Bearsden Road, Glasgow G61 1QH, UK.
Rodrigo
R
Gutierrez-Quintana
College of Medical and Dental Sciences, University of Birmingham, Birmingham B15 2TT, UK.
Alamin
A
Mohammed
UCL School of Pharmacy, 29-39 Brunswick Square, London WC1N 1AX, UK.
Ahad A
AA
Rahim
College of Medical and Dental Sciences, University of Birmingham, Birmingham B15 2TT, UK.
Richard I
RI
Tuxworth
Cardiff School of Biosciences, Cardiff University, The Sir Martin Evans Building, Museum Avenue, Cardiff CF10 3AX, UK.
Kim
K
Wager
MRC Laboratory for Molecular Cell Biology, University College of London, Gower Street, London WC1E 6BT, UK. Electronic address: meb.bond@gmail.com.
Michael
M
Bond
Model systems; NCL; Neurodegeneration; Neuronal ceroid lipofuscinosis