Efficient in vivo base editing via single adeno-associated viruses with size-optimized genomes encoding compact adenine base editors

The viral delivery of base editors has been complicated by their size and by the limited packaging capacity of adeno-associated viruses (AAVs). Typically, dual-AAV approaches based on trans-splicing inteins have been used. Here we show that, compared with dual-AAV systems, AAVs with size-optimized g...

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Detalles Bibliográficos
Autores principales: Davis, Jessie R., Wang, Xiao, Witte, Isaac P., Huang, Tony P., Levy, Jonathan M., Raguram, Aditya, Banskota, Samagya, Seidah, Nabil G., Musunuru, Kiran, Liu, David R.
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/PMC9652153/
https://www.ncbi.nlm.nih.gov/pubmed/35902773
http://dx.doi.org/10.1038/s41551-022-00911-4

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