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Rare disease Models
Group
Head of Group
The research group of Rare Disease Models aims to develop the next generation disease models for neuromuscular disorders (NMD), a wide range of rare diseases affecting muscles, nerves, neuromuscular junctions, and or the brain.
We 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 (base editing) to generate 2D or 3D neuron or muscle models, in order to uncover the disease mechanisms or develop efficient treatment such as CRISPR gene therapy.
