G.P.27: Latest updates to the MDA monoclonal antibody resource for neuromuscular disorders

Abstract

Specific antibodies are vital research tools in the fight against neuromuscular disease, but no single antibody can perform all the necessary functions. Over a period of 20 years, we have produced large numbers of well-characterized antibodies for studies of the most common neuromuscular diseases (Duchenne/Becker and Emery-Dreifuss muscular dystrophies, spinal muscular atrophy and myotonic dystrophy). This MDA Monoclonal Antibody (mAb) resource currently contains over 350 different mAbs, including 150 exon-specific dystrophin mAbs. The antibody panels are currently used by the research community (a) for assessing the outcome of gene therapy, cell therapy, drug therapy or oligonucleotide therapy trials (e.g. dystrophin switch-on or alternative splicing) (b) for animal or cellular model systems to look for novel drug treatments (e.g. SMN or utrophin up-regulation) or novel approaches to gene/protein replacement, and (c) for basic research into understanding disease pathogenesis with the eventual aim of discovering novel treatment targets (e.g. muscleblind and msh3 in myotonic dystrophy). All mAbs are freely available for academic research, subject to an agreed form of acknowledgement in any publications. We now describe the most recent additions to the resource, including new mAbs against nebulin and nesprins 1 and 2. Technical advice freely available from the resource, including datasheets and protocols for producing and applying mAbs, will also be presented. The resource website can be found at www.glennmorris.org.uk/mabs.htm .