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专利名称：Composition and method for introduction of
RNA interference sequences into targetedcells and tissues
发明人：Michael R. Simon申请号：US11126551申请日：20050511
公开号：US20050260214A1公开日：20051124
摘要：A composition and method are provided by which double-stranded RNAcontaining small interfering RNA nucleotide sequences is introduced into specific cellsand tissues for the purpose of inhibiting gene expression and protein production in thosecells and tissues. Intracellular introduction of the small interfering RNA nucleotidesequences is accomplished by the internalization of a target cell specific ligand to whichthe double-stranded RNA containing a small interfering RNA nucleotide sequence isconjugated. The ligand is specific to a unique target cell surface antigen. The ligand iseither spontaneously internalized after binding to the cell surface antigen. Internalizationis also facilitated by the binding of an RNA binding protein to the double-stranded RNA.If the unique cell surface antigen is not naturally internalized after binding to its ligand,internalization is promoted by the incorporation of an arginine-rich peptide, or othermembrane permeable peptide, into the structure of the ligand or attachment of such apeptide to the ligand. The composition and method are practiced in whole livingmammals, as well as cells living in tissue culture. The dsRNA is then hydrolyzed by Dicer,an RNAse III-like ribonuclease, thereby releasing siRNA which silences the target gene.
Inhibition is nucleotide sequence specific and depends upon sequence identity of smallinterfering RNA with the target nucleic acid.
申请人：Michael R. Simon
地址：Ann Arbor MI US
国籍：US
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