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

• Autori principali: Wenwei Shao (Autore), Junjiang Sun (Autore), Xiaojing Chen (Autore), Amanda Dobbins (Autore), Elizabeth P. Merricks (Autore), R. Jude Samulski (Autore), Timothy C. Nichols (Autore), Chengwen Li (Autore)
• Natura: Libro
• Pubblicazione: Frontiers Media S.A., 2022-01-01T00:00:00Z.
• Soggetti: article
• Accesso online: Connect to this object online.

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...