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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מידע ביבליוגרפי

Principais autores:	Wenjia Yu, Yujie Wang, Siwei Li, Yingcai Dai, Yucheng Li, Xinyue Zhang, Bo Li, Siriguleng Qian, Xueli Zhang, Changhao Bi
פורמט:	Artigo
שפה:	Inglês
יצא לאור:	KeAi Communications Co., Ltd. 2025-09-01
סדרה:	Synthetic and Systems Biotechnology
נושאים:	Base editors (BEs) Adeno-associated virus (AAV) Trans-splicing Dual-AAV systems Cas9 split site AAV production titer
גישה מקוונת:	http://www.sciencedirect.com/science/article/pii/S2405805X25000420
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