By Stephen Parmley, Senior Writer
Despite the recent gains in gene therapy, the main system for delivering genes - adeno-associated virus (AAV) vectors - still cannot target tissues of interest efficiently and leave other tissues untouched. A German group has engineered a chimeric form of AAV for customized targeting, based on DARPin antibody-like scaffolds that direct the vectors to cells expressing the antigen of interest. To move to the clinic, the team will need the capabilities and backing of their collaborators at Molecular Partners AG.
The strategy based on designed ankyrin repeat proteins (DARPins) is a departure from the more common approach of trying to solve the targeting problem by searching for rare serotypes of AAV with natural preferences for specific tissues.