Novel Genome-Editing-Based Approaches to Treat Motor Neuron Diseases: Promises and Challenges

By
Justin.choi
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0
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Scientists review genome editing approaches aimed either at correcting the disease-causing mutations or at modulating the expression of genetic modifiers, e.g. by repairing SOD1 mutations or SMN2 splicing defect, or deleting C9orf72 expanded repeats.
[Molecular Therapy]
Miccio, A., Antoniou, P., Ciura, S., & Kabashi, E. (2021). Novel genome-editing-based approaches to treat motor neuron diseases: promises and challenges. Molecular Therapy, 0(0). https://doi.org/10.1016/j.ymthe.2021.04.003 Cite
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