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.
Investigating Therapeutic Options to Increase STMN2 Levels
Researchers at the Sean M. Healey & AMG Center for ALS, along with their colleagues, demonstrated that pharmacological compounds and gene therapy that increase the levels of STMN2 can restore the ability of neurons to grow axons after injury.
