The goal of gene therapy for underlying genetic diseases such as muscular dystrophy or hemophilia is long-term gene expression. Yet there are gene therapy applications in which only short-term expression of a transgene is desirable, such as in the treatment of time-limited diseases such as autoimmunity, infection, or cancer. Another application would be a circumstance in which the gene therapy might cause unwanted side effects, and there would be a desire to inactivate the transgene such as CRISPR/Cas9. Current methods do not allow for controlled expression of trans gene in human patients.
TransSkip is a novel method for controlling transgene expression in gene therapy applications. TransSkip technology enables transgene expression to be dependent on administration of drug. Following a single dose of the viral vector, administration of the drug activates gene expression, and upon discontinuation of the drug expression reverts to being inactive.