Engineering of BZ transposase and transposon donor vector for enhanced efficiency and safety in gene delivery applications.

ABSTRACT: Transposons, as non-viral vectors, provide an efficient and secure method for stable gene delivery and have been successfully applied in human gene therapies. The engineering of transposase has significantly improved the efficiency of various transposon systems, including chimeric antigen receptor (CAR)-T cell engineering. In this study, multiple engineering strategies were implemented to enhance the efficiency and safety of the Baize (BZ) transposon system, which was derived from the ZB (the wild-type BZ, BZwt) and has been proven to be an effective tool for genetic manipulation in vertebrates. Through designed engineering and combinatorial mutagenesis in vitro, several hyperactive BZ transposase variants with higher transposition activity, cargo capacity, and integration safety were deve

SUBMITTER: Wang S 

PROVIDER: S-EPMC12455594 | biostudies-literature | 2025 Sep

REPOSITORIES: biostudies-literature