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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...
Autores principales: | Anderson, Cynthia D., Arthur, Jennifer Ataam, Zhang, Yuan, Bharucha, Nike, Karakikes, Ioannis, Shohet, Ralph V. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
American Society of Gene & Cell Therapy
2023
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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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