AAV Vector Transduction Restriction and Attenuated Toxicity in hESCs via a Rationally Designed Inverted Terminal Repeat
Liujiang Song, Tomoko Hasegawa, Nolan J Brown, Jacquelyn J. Bower, Richard Jude Samulski, Matthew Louis Hirsch
This study evaluates a rationally designed ITR that reduces toxicity and transduction restriction in hESCs while maintaining gene delivery efficiency.
Nucleic Acids Research · Jan 7, 2025Peer-reviewedMolecular Biology