New Pathway Offers Hope for Early ALS Intervention
Researchers at UC San Diego have identified a mechanism that may trigger the early stages of ALS, involving the mislocalization of proteins like CHMP7, which leads to neuron degeneration. By targeting the RNA-binding protein SmD1, they were able to prevent this mislocalization, potentially preserving neuron function. This discovery could lead to therapies that halt ALS progression before significant neurodegeneration occurs, with drugs like risdiplam showing promise in stabilizing neuron integrity.