Inhibition of gene expression by delivery of specially selected double stranded or other forms of small interfering RNA precursors enabling the formation of small interfering RNA in vivo and in vitro

摘要

Inhibiting expression of a target gene by RNA interference, where the target region is selected from that which has another complementary or near complementary region(s) in the same target gene so that a natural hairpin structure of a dsRNA or multiples of the same or different hairpin dsRNA could have been formed. Inhibiting expression of a target gene in a cell comprises introducing a double stranded or other forms of RNA (dsRNA and other small interfering RNA precursors siRNAp) enabling siRNA, where the dsRNA and siRNAp comprise, in most cases, a double stranded structure or a single stranded hairpin structure with an identical or near identical sequence as compared to a region of the target gene. The region selected for inhibition, will be a region that should have another complementary or near complementary region(s) in the same target gene so that a natural hairpin structure of a dsRNA or multiples of the same or different hairpin dsRNA could have been formed, at least in theory, de novo from that particular targeted gene. For practical purposes, the best match sometimes can only be found for the first 4-12 nucleotides or so since the chance of a match is exponentially more difficult after that number. The match may not be restricted to start from the first 4-12 nucleotides but may start and end from and in any position. This concept is applied for the selection of the right sequences for dsRNA and siRNAp to enable target gene silencing. ACTIVITY : Respiratory-Gen.; Anti-HIV; Cytostatic; Viricide; Antibacterial; Antifungal; Antiparasitic. No biological data given. MECHANISM OF ACTION : Gene therapy; RNAi; RNA interference.