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

• Hoofdauteurs: Nicholas M. Kanaan (Auteur), Rhyomi C. Sellnow (Auteur), Sanford L. Boye (Auteur), Ben Coberly (Auteur), Antonette Bennett (Auteur), Mavis Agbandje-McKenna (Auteur), Caryl E. Sortwell (Auteur), William W. Hauswirth (Auteur), Shannon E. Boye (Auteur), Fredric P. Manfredsson (Auteur)
• Formaat: Boek
• Gepubliceerd in: Elsevier, 2017-09-01T00:00:00Z.
• Onderwerpen: article
• Online toegang: Connect to this object online.

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...