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Ian E. Alexander

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Open access Aug 2026

Functional evaluation of a natural AAV capsid liver targeting motif in human hepatocytes

The results showed the potential of a combinatorial AAV library for model validation and revealed the human microliver platform-PEG as a reliable system for the development of AAV therapeutics.

Carmen Unzu, Amanda X. Chen, Liliana Mancio-Silva et al. · 0 citations
Open access Jul 2026

Identification of AAV Vector Integration Sites in Primary Human and Macaque Hepatocytes in vivo, and Analysis of Oncogenic Risk.

RAAV integration patterns in primary human hepatocytes xenografted into FRG mouse livers and in hepatocytes from cynomolgus macaques following systemic rAAV administration support a low oncogenic risk profile for the evaluated vector while reinforcing the value of direct human liver integration analyses to refine risk assessment and guide the development of safer gene therapy platforms.

S. Scott, C. Hallwirth, Natsuki Sasaki et al. · 0 citations