Identifying novel therapeutic targets related to TDP-43 loss
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Despite the diversity of ALS, almost all patients have one thing in common: the protein TDP-43 stops performing its normal functions in the nucleus, leading to disrupted splicing and decreased expression of many genes.
The Impact of Gene Fusions on ALS Disease Progression
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We predict that ALS-specific gene fusions may improve many aspects of clinical care as already
demonstrated in the field of oncology and are now working to understand how the fusions effect ALSdisease progression.