Viral vectors such as adeno-associated virus (AAV) hold tremendous promise for treating genetic diseases. However, candidate AAV therapies have struggled to overcome dose-limiting immune responses, the underlying mechanisms for which are still unclear. We are investigating the cell types and pathways responsible for AAV-induced inflammation, with the goal of improving the safety and durability of gene therapy.
Precise cell type targeting is critical for many gene therapy applications, yet engineering vectors that drive strong and specific expression remains challenging. Using multi-omics and artificial intelligence, we are designing new sequences to control gene activity, enabling robust expression in disease-relevant cell types while minimizing off-target effects.
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RNA therapies have demonstrated success as vaccines and gene-silencing drugs, but their potential for gene editing and protein replacement is still being realized. We are exploring emerging RNA modalities and delivery systems with the aim of translating them into therapeutics and expanding the toolkit for non-viral gene therapy.