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. 2016 May 10;8(6):592–594. doi: 10.15252/emmm.201606407

Figure 1. Selection and functional validation of brain‐targeting AAVs.

Figure 1

Libraries of AAV2 vectors expressing random peptide inserts on their capsid proteins were injected intravenously in mice. The population of AAVs enriched in the brain was subjected to reiterated selection rounds, leading to the identification of a candidate lead viral vector (AAV‐BR1), which was exploited to selectively deliver a therapeutic transgene (AAV‐BR1‐NEMO) to mouse models of incontinentia pigmenti, a severe human genetic disorder of brain vasculature.