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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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Detalles Bibliográficos
Autores principales: Kanaan, Nicholas M., Sellnow, Rhyomi C., Boye, Sanford L., Coberly, Ben, Bennett, Antonette, Agbandje-McKenna, Mavis, Sortwell, Caryl E., Hauswirth, William W., Boye, Shannon E., Manfredsson, Fredric P.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2017
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5503098/
https://www.ncbi.nlm.nih.gov/pubmed/28918020
http://dx.doi.org/10.1016/j.omtn.2017.06.011