Gene therapy vectors

Gene therapy aims to correct health conditions caused by DNA errors, which are often inherited, lifelong, and debilitating. The delivery of a healthy copy of the erroneous information to affected cells typically uses virus-derived vectors, with adeno-associated viruses (AAVs) being the preferred choice due to their long-term efficacy and minimal immune response.

However, natural AAVs lack the specificity to target only affected cells, leading to side effects and limiting their broader application.

This project aims to overcome current AAV limitations by developing next-generation AAV-based vectors with unprecedented selectivity for affected cell populations. Our retargeting technology can address various genetic conditions affecting different cell-types and organs, not limited to a single application. This innovative approach promises to revolutionise gene therapy by enhancing treatment accuracy and control, potentially leading to more effective therapies for genetic disorders and improving the lives of countless individuals through precision medicine.