Gene Replacement Therapy for Tauopathies

Principal Investigator: Dr. Rachel Baily
Center for Alzheimer’s and Neurodegenerative Diseases, UT Southwestern Medical Center, Dallas, TX

The abnormal misfolding and aggregation of proteins are common pathologic features of most neurodegenerative diseases, including tauopathies, with a toxic gain and/or loss of normal protein function being an underlying mechanism of neuronal death. This research will test a gene therapy approach for tauopathies to achieve silencing of the inherent toxic protein in combination with restoration of normal protein function by delivering an aggregate resistant gene. If successful, this could establish a novel therapeutic approach that can be applied to PSP, CBD and a number of protein-linked neurodegenerative diseases.