Cross-species evolution of a highly potent AAV variant for therapeutic gene transfer and genome editing

Recombinant adeno-associated viral (AAV) vectors are a promising gene delivery platform, but ongoing clinical trials continue to highlight a relatively narrow therapeutic window. Effective clinical translation is confounded, at least in part, by differences in AAV biology across animal species. Here...

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Detalles Bibliográficos
Autores principales: Gonzalez, Trevor J., Simon, Katherine E., Blondel, Leo O., Fanous, Marco M., Roger, Angela L., Maysonet, Maribel Santiago, Devlin, Garth W., Smith, Timothy J., Oh, Daniel K., Havlik, L. Patrick, Castellanos Rivera, Ruth M., Piedrahita, Jorge A., ElMallah, Mai K., Gersbach, Charles A., Asokan, Aravind
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2022
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9548504/
https://www.ncbi.nlm.nih.gov/pubmed/36210364
http://dx.doi.org/10.1038/s41467-022-33745-4

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