Zuoshang Xu, PhD & John Landers, PhD
Modeling ALS-Associated Cytoskeletal Mutations in iPSC and Drosophila
Despite the recent discovery of a plethora of new causal genes for ALS, the available mammalian model for mechanistic and therapeutic studies is still limited to transgenic mice expressing mutant SOD1, which has limited further progress in understanding the disease mechanism and therapy. This proposal seeks to change this condition by analyzing transgenic mice expressing mutant profilin 1, another gene linked to ALS. By comparative studies with mutant SOD1 transgenic mice, we will understand generalizable mechanisms as well as specific differences between these models. We will also establish this model as an alternative to the mutant SOD1 model for therapeutic trials in animals.