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Chimeric Mice Engrafted With Canine Hepatocytes Exhibits Similar AAV Transduction Efficiency to Hemophilia B Dog

Adeno-associated virus (AAV) mediated gene therapy has been successfully applied in clinical trials, including hemophilia. Novel AAV vectors have been developed with enhanced transduction and specific tissue tropism. Considering the difference in efficacy of AAV transduction between animal models an...

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Principais autores: Wenwei Shao, Junjiang Sun, Xiaojing Chen, Amanda Dobbins, Elizabeth P. Merricks, R. Jude Samulski, Timothy C. Nichols, Chengwen Li
Formato: Artigo
Idioma:Inglês
Publicado em: Frontiers Media S.A. 2022-01-01
coleção:Frontiers in Pharmacology
Assuntos:
liver transduction
chimeric mice
hemophilia B
canine hepatocytes
AAV (Adeno-associated virus)
Acesso em linha:https://www.frontiersin.org/articles/10.3389/fphar.2022.815317/full
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