Engineered AAVS for CNS Transduction and Peripheral Organ De-Targeting across Species after Systemic Delivery

Abstract

In recent years, we have witnessed the development and clinical use of gene therapies at an accelerated pace. The option to introduce, silence, or edit genes greatly increases the therapeutic avenues for a variety of diseases, with adeno-associated viruses (AAVs) being the vehicle of choice due to their low immunogenicity, stable expression, and strong clinical safety record. While the route of delivery has traditionally been localized, systemic administration via the blood is an option in cases where direct injection is impractical or widespread cell populations are affected. However, the naturally occurring AAV serotypes have evolved to broadly infect tissue, an undesirable characteristic for targeting therapeutics to distinct cells.

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