Identifying novel therapeutic targets related to TDP-43 loss
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
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.