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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Detalhes bibliográficos
Publicado no:Mol Ther Nucleic Acids
Main Authors: 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: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2017
Assuntos:
Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5503098/
https://ncbi.nlm.nih.gov/pubmed/28918020
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtn.2017.06.011
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