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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...
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. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
American Society of Gene & Cell Therapy
2017
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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 |
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