David Corey and his team at Harvard Medical School are starting to investigate a reduced Pcdh15b gene (mini-genes). By shrinking the protein of interest of USH 1F, it can be mini-packaged in an AAV vector and then injected into the inner ear and / or retina.
For this study “gene therapy minigenes”, the mice will be used as an animal model to investigate the effect on the mice’s hearing.
A zebrafish will be used as an animal model for research into the consequences of vision.
Jen Philips and Monte Westerfield have developed a zebrafish model Pcdh15b using gene-editing with the Crispr / Cas9 technique.