One-and-done gene therapy wipes out Parkinson's symptoms in mouse models

A researcher at the University of San Diego, California (USCD) made an accidental discovery several years back after he silenced a gene called PTB in mouse fibroblasts, cells in connective tissue. Within weeks, almost all of the fibroblasts were gone, and the rest had transformed into neurons.

Now, researchers in the same lab are applying that discovery to Parkinson’s disease in the hopes of creating a one-time gene therapy to replace the dopamine-producing neurons that are lost to the disease.