A multiplexed barcode approach to simultaneously evaluate gene delivery by adeno-associated virus capsid variants in nonhuman primates.

Daniel Stone Nadja MeumannAnne-Sophie KuhlmannChristopher W Peterson Hong Xie Pavitra Roychoudhury Michelle A LoprienoXuan-Khang VuDaniel E StronginElizabeth J KenkelAnoria HaickLaurence StenslandWillimark M ObenzaJacob ParrottVeronica NelsonRobert D MurnaneMeei-Li W Huang Martine Aubert Hans-Peter Kiem Hildegard Büning Keith R Jerome

Published in: Hepatology communications (2023)

This method offers a robust approach to AAV vector optimization that can be used to identify and validate AAV vectors for gene delivery to potentially any anatomical site or cell type.

Keyphrases

gene therapy
copy number
single cell