A Novel Triple-Mutant AAV6 Capsid Induces Rapid and Potent Transgene Expression in the Muscle and Respiratory Tract of Mice

Gene therapy for the treatment of genetic disorders has demonstrated considerable therapeutic success in clinical trials. Among the most effective and commonly used gene delivery vectors are those based on adeno-associated virus (AAV). Despite these advances in clinical gene therapy, further improve...

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Bibliographic Details
Main Authors: Laura P. van Lieshout, Jakob M. Domm, Tara N. Rindler, Kathy L. Frost, Debra L. Sorensen, Sarah J. Medina, Stephanie A. Booth, James P. Bridges, Sarah K. Wootton
Format: Article
Language:English
Published: Elsevier 2018-06-01
Series:Molecular Therapy: Methods & Clinical Development
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050118300433