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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Bibliographic Details

Published in:	Molecular Therapy: Nucleic Acids
Main Authors:	Cynthia D. Anderson, Jennifer Ataam Arthur, Yuan Zhang, Nike Bharucha, Ioannis Karakikes, Ralph V. Shohet
Format:	Article
Language:	English
Published:	Elsevier 2023-09-01
Subjects:	MT: Delivery strategies base editing hepatocytes focused ultrasound microbubbles ultrasound targeted microbubble destruction
Online Access:	http://www.sciencedirect.com/science/article/pii/S2162253123002056