We are editing DNA in motor neurons to develop a treatment for a currently incurable genetic neuropathy.
The power of CRISPR genome editing provides the exciting potential to cure motor neuron diseases with no available treatment. Dominant negative mutations cause Charcot-Marie-Tooth disease, a motor neuron disorder that leads to progressive loss of the neurons that control leg and arm muscles. We are optimizing the CRISPR-Cas9 genome engineering system to efficiently correct the disease-causing mutations in motor neurons while avoiding unintended genetic changes. We grow motor neurons from the patient’s stem cells (iPSCs), allowing us to test editing methods in the patient’s own cells before a clinical trial. We are exploring delivery methods to deliver CRISPR editing molecules directly into motor neurons to fix the disease-causing mutation. These proof-of-concept studies will provide the first steps toward potential therapeutic editing in motor neurons.
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