Peptide, Complex of Peptide and siRNA, and Methods of Use Thereof

Abstract

A series of peptides and a peptide-siRNA complex are disclosed, wherein the peptide based complex effectively enhances delivery of siRNA molecules into the cells and release of siRNA in the cell, and improves siRNA mediated gene silencing efficiency of cellular targets. Pharmaceutical compositions that include the complex, as well as a use of the complex in the gene therapy field, are also disclosed.

What is claimed is: 
     
         1 . A peptide comprising a hydrophobic part and a hydrophilic part, wherein the hydrophilic part comprises a positively charged amino acid residue, wherein the peptide is CH 3 (CH 2 ) 16 CONH—B—CONH 2 , and wherein B is SEQ ID NO:4 or 5. 
     
     
         2 . A complex comprising:
 a cargo molecule; and   a peptide comprising a hydrophobic part and a hydrophilic part, wherein the hydrophilic part comprises a positively charged amino acid residue, wherein the peptide is CH 3 (CH 2 ) 16 CONH—B—CONH 2 , and wherein B is SEQ ID NO:4 or 5.   
     
     
         3 . The complex according to  claim 2 , wherein the cargo molecule and the peptide are coupled non-covalently. 
     
     
         4 . The complex according to  claim 2 , wherein the particle size of the complex is in a range of from about 20 nm to about 999 nm. 
     
     
         5 . The complex according to  claim 2 , wherein the cargo molecule is a nucleic acid. 
     
     
         6 . The complex according to  claim 5 , wherein the nucleic acid is a short interfering RNA (siRNA). 
     
     
         7 . The complex according to  claim 6 , wherein the siRNA is complexed with the peptide at a molar ratio within a range of from about 1:1 to about 80:1. 
     
     
         8 . The complex according to  claim 6 , wherein the siRNA is complexed with the peptide at a molar ratio of about 20:1. 
     
     
         9 . The complex according to  claim 6 , wherein the siRNA is complexed with the peptide at a molar ratio of about 40:1. 
     
     
         10 . The complex according to  claim 6 , wherein the siRNA is complexed with the peptide at a molar ratio of about 60:1. 
     
     
         11 . A pharmaceutical composition containing a complex comprising:
 a cargo molecule; and   a peptide comprising a hydrophobic part and a hydrophilic part, wherein the hydrophilic part comprises a positively charged amino acid residue, wherein the peptide is CH 3 (CH 2 ) 16 CONH—B—CONH 2 , and wherein B is SEQ ID NO:4 or 5.   
     
     
         12 . A kit used to deliver drugs to a patient, wherein the kit comprises:
 a pharmaceutical composition comprising a complex comprising:
 a cargo molecule; and 
 a peptide comprising a hydrophobic part and a hydrophilic part, wherein the hydrophilic part comprises a positively charged amino acid residue, wherein the peptide is CH 3 (CH 2 ) 16 CONH—B—CONH 2 , and wherein B is SEQ ID NO:4 or 5; 
   one or more electrolytes;   one or more buffers;   a delivery device; and   a container adapted to mix one or more other agents together.   
     
     
         13 . The kit according to  claim 12 , wherein the cargo molecule and the peptide are coupled non-covalently. 
     
     
         14 . The kit according to  claim 12 , wherein the particle size of the complex is in a range of from about 20 nm to about 999 nm. 
     
     
         15 . The kit according to  claim 12 , wherein the cargo molecule is a nucleic acid. 
     
     
         16 . The kit according to  claim 15 , wherein the nucleic acid is a short interfering RNA (siRNA). 
     
     
         17 . The kit according to  claim 16 , wherein the siRNA is complexed with the peptide at a molar ratio within a range of from about 1:1 to about 80:1. 
     
     
         18 . The kit according to  claim 16 , wherein the siRNA is complexed with the peptide at a molar ratio in a range of from about 20:1 to about 60:1. 
     
     
         19 . The kit according to  claim 16 , wherein the siRNA is complexed with the peptide at a molar ratio of about 20:1 or about 40:1. 
     
     
         20 . The kit according to  claim 16 , wherein the siRNA is complexed with the peptide at a molar ratio of about 60:1.