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Cytosine and adenine base editing of the brain, liver, retina, heart and skeletal muscle of mice via adeno-associated viruses

The success of base editors for the study and treatment of genetic diseases depends on the ability to deliver them in vivo to the relevant cell types. Delivery via adeno-associated viruses (AAVs) is limited by AAV-packaging capacity, which precludes the use of full-length base editors. Here, we repo...

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Detalles Bibliográficos
Autores principales: Levy, Jonathan M., Yeh, Wei-Hsi, Pendse, Nachiket, Davis, Jessie R., Hennessey, Erin, Butcher, Rossano, Koblan, Luke W., Comander, Jason, Liu, Qin, Liu, David R.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6980783/
https://www.ncbi.nlm.nih.gov/pubmed/31937940
http://dx.doi.org/10.1038/s41551-019-0501-5