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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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Principais autores: Cynthia D. Anderson, Jennifer Ataam Arthur, Yuan Zhang, Nike Bharucha, Ioannis Karakikes, Ralph V. Shohet
Format: Artigo
Jezik:Inglês
Izdano: Elsevier 2023-09-01
Serija:Molecular Therapy: Nucleic Acids
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Online dostop:http://www.sciencedirect.com/science/article/pii/S2162253123002056
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