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AAV vectors with enhancer-controlled ITR promoters

Zheng, Y.; Yang, L.; Zhou, K.; Wang, Q.; Li, T.; Wu, J.; Qin, J.; Gao, F.; Zhou, P.; Lu, T.; Cao, Y.; Hua, Y.; Guo, Y.; Li, Y.

2025-12-05 bioengineering
10.64898/2025.12.03.692098 bioRxiv
Show abstract

Adeno-associated virus (AAV) is limited by its packaging capacity and the unwanted promoter activity of inverted terminal repeats (ITRs). Here we utilized mini-enhancers to regulate ITR promoters and drive AAV cargo expression in the absence of the canonical promoters, which not only solved the ITR issue but also released more payload for the cargo. As an example, this new design successfully enabled robust, tissue-specific SpCas9 gene editing via a single AAV, which otherwise requires a dual-AAV system due to the large size of SpCas9.

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"Similar papers" are the closest papers from that journal in the model's embedding space. They show what the match is built on, but the ranking comes mostly from a classifier over the whole training set, not from these examples alone.