University of Pittsburgh School of Medicine is working on a technology to help other researchers determine what the best gene delivery system could be for their genetic treatment research. scAAvengr provides quick turnaround time to ensure efficient gene treatment delivery. To target specific cells, therapeutic genes need to be "carried", usually by viral vectors, and then released once they reach their targets. The traditional method of choosing a vector that is both specific enough to target the desired cell and also able to release the gene is very time consuming. With the use of this technology platform, researchers will be able to determine an efficient gene delivery system faster and hopefully lead to more therapies entering clinical trials.
What this means for Usher syndrome: If this platform is able to help researchers choose delivery systems to target retinal cells specifically, it may also help researchers choose delivery systems that can target cells affected in Usher syndrome.