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Non-viral in vivo cytidine base editing in hepatocytes using focused ultrasound targeted microbubbles

CRISPR-Cas9-based genome editing technologies, such as base editing, have the potential for clinical translation, but delivering nucleic acids into target cells in vivo is a major obstacle. Viral vectors are widely used but come with safety concerns, while current non-viral methods are limited by lo...

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Detalles Bibliográficos
Autores principales: Anderson, Cynthia D., Arthur, Jennifer Ataam, Zhang, Yuan, Bharucha, Nike, Karakikes, Ioannis, Shohet, Ralph V.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10468349/
https://www.ncbi.nlm.nih.gov/pubmed/37662969
http://dx.doi.org/10.1016/j.omtn.2023.07.032

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