Rare Disease Models

The research group of Dr. Haicui Wang aims to develop the next generation disease cell models for neuromuscular disorders (NMD), a wide range of rare diseases affecting muscles, nerves, neuromuscular junctions, and or the brain. 
More and more disease-causing genes have been discovered with development of the next generation sequencing, yet the underlying disease mechanisms remain unclear and there is lack of efficient treatment for most cases. Disease specific, functional related in vitro models are at urgent need. 
Along with the previous models from limited and valuable patient primary cells, the next generation cell models will take the good use of unlimited human induced pluripotent stem cell (iPSC) lines or other continuous cell lines, in combination with the state of the art CRISPR techniques to generate the disease genetic background.