Cell and Animal Based Research
Supporting preclinical scientists and trainees is a key focus of the network, and together the animal and cell-based research tasks work towards key outputs to standardize and make accessible specialist scientific protocols and provide research training and skill development in preclinical modelling and bioinformatics.
Cell-Based Neuromuscular Research
Recent advances in cell-based research and human iPSC-derived 3D models have delineated key contributions of muscle stem cells in regeneration, as well as the role of different cell populations and their expression patterns in muscle degeneration. In addition to wet lab techniques, cell-based researchers increasingly require bioinformatics skills to interpret -omics data. We will work to provide access to training in these cutting-edge techniques and create a broader skills base across the NMD research community by supporting individualized training visits to upskill NMD trainees.
Animal-Based Neuromuscular Research
Animal models, particularly mice and zebrafish, are powerful tools for understanding and treating NMDs. They enable the effects of altering gene and variant expression to be studied, and can be used to test the safety and efficacy of genetic therapies prior to human trials. NMD researchers need standardized experimental protocols for knowledge exchange and experimental reproducibility between labs. While these exist for some commonly used models, more specific protocols are usually confined to individual labs, and we will work towards the standardization and online publishing of these research protocols.