Novel platforms for studying AAV subcellular biology & the design of improved gene therapy vectors KU Leuven
Adeno-associated virus (AAV) gene therapy is currently experiencing a renaissance thanks to a number of successful clinical trials. However, vector doses required to achieve therapeutic effect are high and some tissues remain difficult targets for efficient transduction. In general, gene therapy strategies directed at the brain of patients with inherited neurological or neurodegenerative disorders have shown limited efficacy. One of the main ...