A humanized mouse model for adeno-associated viral gene therapy

Clinical translation of AAV-mediated gene therapy requires preclinical development across different experimental models, often confounded by variable transduction efficiency. Here, we describe a human liver chimeric transgene-free Il2rg −/− /Rag2 −/− /Fah −/− /Aavr −/− (TIRFA) mouse model overcoming...

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Bibliographic Details
Published in:	Nature communications Vol. 15; no. 1; p. 1955
Main Authors:	Barzi, Mercedes, Chen, Tong, Gonzalez, Trevor J., Pankowicz, Francis P., Oh, Seh Hoon, Streff, Helen L., Rosales, Alan, Ma, Yunhan, Collias, Sabrina, Woodfield, Sarah E., Diehl, Anna Mae, Vasudevan, Sanjeev A., Galvan, Thao N., Goss, John, Gersbach, Charles A., Bissig-Choisat, Beatrice, Asokan, Aravind, Bissig, Karl-Dimiter
Format:	Journal Article
Language:	English
Published:	London Nature Publishing Group UK 04-03-2024 Nature Publishing Group Nature Portfolio
Subjects:	13 13/51 14 14/32 42 42/34 631/154/51/201 631/1647/334/1874/345 631/208/1516 631/61/17/1511 64 64/60 Ablation Animal models Animals Dependovirus - genetics Disease Models, Animal Gene therapy Genetic Therapy Hepatocytes Humanities and Social Sciences Humans Liver Liver cancer Mice multidisciplinary RAG2 protein Receptors Science Science (multidisciplinary) Serotypes Teratoma Transduction Transgenes
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