Biology

Engineering viral vectors for acoustically targeted gene delivery

H. R. Li, M. Harb, et al.

Unlocking the potential for targeted gene delivery to the brain, researchers from Caltech and Rice University have engineered novel AAV vectors that significantly enhance ultrasound-targeted gene delivery while minimizing unwanted effects on peripheral organs. This breakthrough promises a major advancement in treating brain diseases and neuroscience research.

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Abstract

Targeted gene delivery to the brain is crucial for neuroscience research and treating brain diseases. While invasive injections are common, focused ultrasound blood-brain barrier opening (FUS-BBBO) offers a noninvasive, site-specific alternative. However, using natural AAV serotypes with FUS-BBBO has limitations in transduction efficiency and causes significant peripheral organ transduction. This study utilizes high-throughput in vivo selection to engineer novel AAV vectors optimized for local neuronal transduction at FUS-BBBO sites. The engineered vectors significantly enhance ultrasound-targeted gene delivery and neuronal tropism while minimizing peripheral transduction, resulting in a more than ten-fold improvement in targeting specificity in two mouse strains.

Publisher

Nature Communications

Published On

Jun 10, 2024

Authors

Hongyi R. Li, Manwal Harb, John E. Heath, James S. Trippett, Mikhail G. Shapiro, Jerzy O. Szablowski

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