Key result
Next-generation sequencing-guided screening of random AAV capsid peptide libraries in a murine model successfully identified a capsid variant that efficiently targets pulmonary vasculature endothelium.
NGS-guided in vivo screening of AAV peptide libraries enables the identification of highly specific vectors for targeted gene delivery, such as to the pulmonary endothelium.
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May advance safer systemic AAV gene therapy; leaves open clinical translation from murine models.
Körbelin et al. (2016) studied this question. Next-generation sequencing-guided screening of random capsid displayed peptide libraries on AAV was evaluated on Identification of target-specific AAV capsid candidates. Next-generation sequencing-guided screening of random AAV capsid peptide libraries in a murine model successfully identified a capsid variant that efficiently targets pulmonary vasculature endothelium.
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