Rationally Engineered AAV Capsids Improve Transduction and Volumetric Spread in the CNS

Adeno-associated virus (AAV) is the most common vector for clinical gene therapy of the CNS. This popularity originates from a high safety record and the longevity of transgene expression in neurons. Nevertheless, clinical efficacy for CNS indications is lacking, and one reason for this is the relat...

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Bibliographic Details
Main Authors: Nicholas M. Kanaan, Rhyomi C. Sellnow, Sanford L. Boye, Ben Coberly, Antonette Bennett, Mavis Agbandje-McKenna, Caryl E. Sortwell, William W. Hauswirth, Shannon E. Boye, Fredric P. Manfredsson
Format: Article
Language:English
Published: Elsevier 2017-09-01
Series:Molecular Therapy: Nucleic Acids
Online Access:http://www.sciencedirect.com/science/article/pii/S216225311730197X