Generation of novel AAV serotypes with enhanced infectivity, specificity, and lower toxicity via AAV capsid engineering platform

AAV-based gene therapies pose a number of challenges to developers, including ineffective uptake to target tissues, off-target effects and toxicity, and virus neutralization. Therefore, naturally occurring AAV serotypes cannot fulfill all the requirements of targeted gene therapy. Capsid engineering can provide a solution to this issue by enabling successful development of novel AAV serotypes with enhanced specificity, infectivity, and reduced toxicity. In this article, a capsid engineering platform is described, along with study data on promising AAV variants generated using this approach.

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