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专利名称：COMPOSITION AND METHOD FOR
INTRODUCTION OF RNA INTERFERENCESEQUENCES INTO TARGETED CELLS ANDTISSUES
发明人：Michael R. Simon申请号：US12917311申请日：20101101
公开号：US20110117088A1公开日：20110519
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 internalization of a target cell specific ligand to which thedouble-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 a peptide into the structure of theligand or attachment of such a peptide to the ligand.
申请人：Michael R. Simon
地址：Ann Arbor MI US
国籍：US
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