This is an image of a section of the mouse brain. In this image, all brain cells are labeled in blue, neurons are colored red, and an advanced approach to treat neurodegenerative diseases is in green and yellow. We wanted to find the right dose to reach a substantial fraction of neurons with our new therapeutics, so we performed an experiment in which we delivered different doses to different mice, and then looked at their brains under the microscope to measure how many neurons we reached at each dose.

Our goal is to test a host of gene therapies simultaneously in a single neurodegenerative brain. This “pooled screen” approach to gene therapy experiments would dramatically accelerate the rate we can test candidate therapeutics, hastening the development of a potential drug.

We reached our goal; determining the right dose to test many possible gene therapies at once in a single mouse brain.