One sees that in all these cases, intervention by genetic engineering to modify a given transcript in the target cell population could be beneficial. This is what proposes the technology of spliceosome‐mediated RNA trans‐splicing by diverting the spliceosome machinery to modify part of the exons of an endogenous transcript, using an artificial RNA brought by gene transfer into the cells to be corrected.
Since finding effective target sites within RNA is a hard work for binding domains design, various experimental methods and computational approaches have been proposed.
CRAWFISH, is a bioinformatics system built to assist researchers in selecting the
best region of interest to design sequences to induce the exon trans‐splicing.
It can be used to restore functional proteine or silence a gene/transcript.