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C9ORF72 protein_ALS and other Neurological diseases
University of Southern California
Small molecule
Neurology, CNS disease
Hit To Lead or Lead Optimization
USC researchers have identified a method of treating ALS by restoring the levels of the C9ORF72 protein. Additional studies have shown that inhibiting the PIKFYVE kinase with inhibitors prevent the degeneration of neurons derived from ALS patients and rescues neurodegeneration in cell models of sporadic ALS. These results demonstrate how stem cell and reprogramming-based approaches can help to identify new therapeutic targets to treat ALS and other neurological diseases.
US 2018-0161335 A1