Aalto OpenLearning

Transposons have emerged as a potential solution for gene therapy, offering advantages over viral vectors. Transposons facilitate the long-term expression of therapeutic genes by incorporating themselves into the genome of the target cell. What once started as basic tools in biomedical research, transposons have now become established therapeutics. At present, the most encouraging transposons for gene therapy are sourced from the wild-type Sleeping Beauty elements. To enhance stable transposition efficiency, newer transposon systems utilize codon optimization and hyper-activating mutations in SB or PB transposases.