Recently a profilin mouse model was generated that displays the hallmarks of neurodegeneration that we see in SOD1 preclinical models. The transgenic is created in mcuh the same way as other preclinical models where the mutated human profilin gene is expressed in the mouse. As a control they created a profilin transgenic with the wild type human profiln gene and there was no phenotype in this model.
this could be an important tool for preclinical drug development providing another animal model of neurodegeneration that has pathophysiological features that resemble ALS. This has been an issue in the field the last 10 years since many recent transgenics with TDP-43, FUS1, and C9ORF do not have progressive neurodegeneration.
