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AAV-CRISPR Gene Editing Is Negated by Pre-existing Immunity to Cas9

Adeno-associated viral (AAV) vectors are a leading candidate for the delivery of CRISPR-Cas9 for therapeutic genome editing in vivo. However, AAV-based delivery involves persistent expression of the Cas9 nuclease, a bacterial protein. Recent studies indicate a high prevalence of neutralizing antibod...

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Detalles Bibliográficos
Autores principales: Li, Ang, Tanner, Mark R., Lee, Ciaran M., Hurley, Ayrea E., De Giorgi, Marco, Jarrett, Kelsey E., Davis, Timothy H., Doerfler, Alexandria M., Bao, Gang, Beeton, Christine, Lagor, William R.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7264438/
https://www.ncbi.nlm.nih.gov/pubmed/32348718
http://dx.doi.org/10.1016/j.ymthe.2020.04.017