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Characterization of the Humanized FRG Mouse Model and Development of an AAV-LK03 Variant with Improved Liver Lobular Biodistribution

Marti Cabanes-Creus,Renina Gale Navarro,Sophia H. Y. Liao,Suzanne Scott,Rodrig Carlessi,Ramon Roca-Pinilla,Maddison Knight,Grober Baltazar,Erhua Zhu,Matthew Jones,Elena Denisenko,Alistair R. R. Forrest,Ian E. Alexander,Janina E. E. Tirnitz-Parker, Lesze Lisowski

Molecular therapy Methods & clinical development（2023）

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关键词
adeno-associated vector,liver lobule,gene therapy,liver zonation,humanized FRG,lobular transduction,single-nucleus RNA sequencing
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