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OwnerJiahong SHAO, Pengcheng TAN +5inventors
InventorsJiahong SHAO, Pengcheng TAN, Xiang WU, Xiaoming ZHAO, Yang LU, Tingjun XUN, Zhenzhen LEI
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Provided are a recombinant adeno-associated virus (rAAV) genome and a single-polarity rAAV (spAAV) vector formed by packaging same. Specifically, provided is an spAAV genome before packaging, two ends of which are both linear open terminuses, wherein one end does not have an inverted terminal repeat (ITR) or a part thereof, and the other end has a truncated ITR (ITRT), and the ITRT does not form a T-shaped hairpin palindromic structure. The spAAV genome is a single-polarity single-stranded DNA. Also provided are an spAAV vector, a method for delivering an exogenous nucleic acid to a cell by using the vector, and a use of the vector in the preparation of a product for gene expression, gene therapy, gene editing or gene regulation.
An unpackaged single polarity recombinant adeno-associated virus (spAAV) genome, having linear, open-ended termini at both ends, with one end lacking an inverted terminal repeat (ITR) or a portion thereof, and the other end having a truncated ITR (ITRt), wherein the ITRt does not form a T-shaped hairpin palindrome structure;
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