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Second Generation AAV Capsids Reprogrammed to Bind Human Transferrin Receptor Are Targeted to the Brain and De-Targeted from the Liver in Human TFRC Knock-In Mice

Ken Y. Chan,Qin Huang, Shan Lou,Casey Keyes,Jason Wu, Nuria R. Botticello-Romero,Qingxia Zheng, Chin-Yen Lin,Jencilin Johnston,Allan Mills,Pamela Brauer,Gabrielle Clouse,Simon Pacouret, John W. Harvey,Thomas Beddow, Jenna K. Hurley,Isabelle G. Tobey,Megan Powell, Catherine P. Pirtle,Albert Chen,Andrew J. Barry,Fatma-Elzahraa Eid,Yujia A. Chan,Benjamin E. Deverman

MOLECULAR THERAPY（2024）

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