A recent biotechnology breakthrough is the organ-on-chip (OOC) technology. Researchers in Germany at the Eberhard Karls University of Tübingen are now combining OOC and human stem cells to make a retina-on-a-chip model which will help determine the effectiveness of different gene therapy vectors. Stem cells are grown by researchers into 3D retinal models, called organoids which have all of the retinal cells. This 3D retina model is then placed into the chamber of a fluidic device (the OOC), allowing researchers to view and feed the tissues of the retina. This can be a model for researchers to analyze the function of AAV vectors to understand their effectiveness.
What this means for Usher syndrome: This retina on a chip creates a new type of model for researchers to study gene therapy effectiveness. With the ability to re-create an exact model of the retina outside the human body, researchers can more easily study AAV vector therapy in addition to other therapy options before getting to clinical trials.