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.
Keywords: artificial intron, posttranscriptional gene silencing, pol-II-directed rna interference (rnai), d-rnai, mirna generation, rna splicing, intron-mediated enhancement (ime)
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