US2020181642A1PendingUtilityA1

Nanoparticle-mediated gene delivery, genomic editing and ligand-targeted modification in various cell populations

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Assignee: RENSSELAER POLYTECH INSTPriority: Sep 23, 2013Filed: Nov 27, 2019Published: Jun 11, 2020
Est. expirySep 23, 2033(~7.2 yrs left)· nominal 20-yr term from priority
Inventors:Shiva Prasad KothaAndre Ronald WatsonVaibhav A. Pandit
C12N 15/88A61K 47/6923A61K 47/6929A61K 47/6455C12N 15/85A61K 48/0041C12N 9/22C12N 2800/95A61P 19/10
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Claims

Abstract

An improved nanoparticle for transfecting cells is provided. The nanoparticle includes a core polyplex and a silica coating on the core polyplex and, optionally, a polymer attached to an outer surface of the silica coating, where the polyplex includes an anionic polymer, a cationic polymer, a cationic polypeptide, and a polynucleotide. Also provided is an improved method of modifying intracellular polynucleotides. The method includes contacting a cell with a nanoparticle that includes a core polyplex and a silica coating on the core polyplex and, optionally, a polymer attached to an outer surface of the silica coating, where the polyplex includes an anionic polymer, a cationic polymer, a cationic polypeptide, and a polynucleotide.

Claims

exact text as granted — not AI-modified
1 . A nanoparticle comprising:
 a core polyplex and a silica coating thereon;   wherein said core polyplex comprises an anionic polymer, a cationic polymer, a cationic polypeptide, and a polynucleotide.   
     
     
         2 . The nanoparticle of  claim 1  wherein the anionic polymer is poly(D-glutamic acid). 
     
     
         3 . The nanoparticle of  claim 1  wherein the cationic polymer is selected from the group consisting of poly(ethylenimine) and poly(L-arginine). 
     
     
         4 . The nanoparticle of  claim 1  wherein the cationic polypeptide is a histone tail peptide. 
     
     
         5 . The nanoparticle of  claim 4  wherein the histone tail peptide is human H3 histone tail peptide. 
     
     
         6 . The nanoparticle of  claim 1  wherein the anionic polymer is poly(D-glutamic acid), the cationic polymer is selected from the group consisting of poly(ethylenimine) and poly(L-arginine), and the cationic polypeptide is a histone tail peptide. 
     
     
         7 . The nanoparticle of  claim 6  wherein the polynucleotide comprises a nucleotide sequence that encodes a nuclease. 
     
     
         8 . The nanoparticle of  claim 7  wherein the nuclease is a TALEN. 
     
     
         9 . The nanoparticle of  claim 8  wherein the TALEN is capable of inducing a break at a site-specific locus of DNA, wherein the break results in a change of expression of a protein encoded by a gene. 
     
     
         10 . The nanoparticle of  claim 9  wherein the change is a decrease and the gene encodes a sclerostin protein. 
     
     
         11 . A nanoparticle of  claim 6 , further comprising a polymer attached to an outer surface of said silica coating. 
     
     
         12 . A nanoparticle of  claim 11 , wherein said polymer attached to an outer surface of said silica coating comprises poly(L-arginine) or a vasoactive endothelial growth factor peptide. 
     
     
         13 . A method of modifying intracellular polynucleotides comprising;
 contacting a cell with a nanoparticle, wherein said nanoparticle comprises a core polyplex and a silica coating thereon;   wherein said core polyplex comprises an anionic polymer, a cationic polymer, a cationic polypeptide, and a polynucleotide.   
     
     
         14 . The method of  claim 13  wherein the anionic polymer is poly(D-glutamic acid). 
     
     
         15 . The method of  claim 13  wherein the cationic polymer is selected from the group consisting of poly(ethylenimine) and poly(L-arginine). 
     
     
         16 . The method of  claim 13  wherein the cationic polypeptide is a histone tail peptide. 
     
     
         17 . The method of  claim 16  wherein the histone tail peptide is human H3 histone tail peptide. 
     
     
         18 . The method of  claim 13  wherein the anionic polymer is poly(D-glutamic acid), the cationic polymer is selected from the group consisting of poly(ethylenimine) and poly(L-arginine), and the cationic polypeptide is a histone tail peptide. 
     
     
         19 . The method of  claim 18  wherein the polynucleotide comprises a nucleotide sequence that encodes a nuclease. 
     
     
         20 . The method of  claim 19  wherein the nuclease is a TALEN. 
     
     
         21 . The method of  claim 20  wherein the TALEN is capable of inducing a break at a site-specific locus of DNA, wherein the break results in a change of expression of a protein encoded by a gene. 
     
     
         22 . The method of  claim 21  wherein the change is a decrease and the gene encodes a sclerostin protein. 
     
     
         23 . The method of  claim 18 , further comprising a polymer attached to an outer surface of said silica coating. 
     
     
         24 . The method of  claim 23 , wherein said polymer attached to an outer surface of said silica coating comprises poly(L-arginine) or a vasoactive endothelial growth factor peptide.

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