Optimized dual-AAV base editor delivery system with enhanced editing efficiency and virion production titer

Base editors (BEs) are a promising tool for precise base conversion in human cells and animals, while the adeno-associated virus (AAV) is the major vector for human gene therapy. However, the size of the DNA cassette required for BE expression exceeds the 4.7 kb packing capacity of the AAV vector, m...

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Autori principali:	Wenjia Yu, Yujie Wang, Siwei Li, Yingcai Dai, Yucheng Li, Xinyue Zhang, Bo Li, Siriguleng Qian, Xueli Zhang, Changhao Bi
Natura:	Artigo
Lingua:	Inglês
Pubblicazione:	KeAi Communications Co., Ltd. 2025-09-01
Serie:	Synthetic and Systems Biotechnology
Soggetti:	Base editors (BEs) Adeno-associated virus (AAV) Trans-splicing Dual-AAV systems Cas9 split site AAV production titer
Accesso online:	http://www.sciencedirect.com/science/article/pii/S2405805X25000420
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