New Drug Design for Gene Therapy - Taking Advantage of Introns

A novel gene modulation system was identified within mammalian introns, regulating intracellular gene transcripts homologous to certain 5'-proximal non-snRNP-binding regions of spliced introns. This gene modulation system can be manipulated for the analysis of gene function and development of gene-specific therapeutics, using artificial introns with 5'-proximal hairpin inserts.