US2004137625A1PendingUtilityA1

Compositions and methods for drug delivery using amphiphile binding molecules

46
Priority: Jan 21, 1999Filed: Jan 12, 2004Published: Jul 15, 2004
Est. expiryJan 21, 2019(expired)· nominal 20-yr term from priority
Inventors:Jon WolffJames HagstromSean D. MonahanVladimir BudkerDavid B. RozemaPaul Slattum
B82Y 5/00C07H 21/00C12N 15/87A61K 47/6951A61K 48/00A61K 48/0025
46
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Claims

Abstract

The present invention relates to the delivery of desired compounds (e.g., nucleic acids) into cells using noncovalent delivery systems which include complexing nucleic acids, amphipathic binding agents, and amphiphiles.

Claims

exact text as granted — not AI-modified
We claim:  
     
         1 . A process for obtaining an expression product by delivering a polynucleotide to a cell, comprising: 
 a. associating an amphiphile binding agent, an amphiphile, and a polynucleotide thereby forming a complex;    b. delivering the complex to the cell; and,    c. expressing the polynucleotide.    
     
     
         2 . The process of  claim 1  wherein the amphiphile binding agent consists of a cyclodextrin.  
     
     
         3 . The process of  claim 1  wherein the amphiphile binding agent is polymeric.  
     
     
         4 . The process of  claim 1  further comprising complexing the polynucleotide with a polycation.  
     
     
         5 . The process of  claim 1  further comprising associating a polyanion in step (a).  
     
     
         6 . The process of  claim 1  wherein the amphiphile consists of a polymer.  
     
     
         7 . The process of  claim 1  wherein the amphiphile consists of an interaction modifier.  
     
     
         8 . The process of  claim 1  wherein the cell is in a mammal.  
     
     
         9 . The process of  claim 1  wherein the polynucleotide consists of DNA.  
     
     
         10 . The process of  claim 1  wherein the polynucleotide consists of a gene.  
     
     
         11 . A complex for delivering and expressing DNA in a mammal, comprising: an amphiphile binding agent, an amphiphile, and DNA in complex.  
     
     
         12 . The complex of  claim 11  wherein the amphiphile is attached to the DNA.  
     
     
         13 . The complex of  claim 12  wherein the amphiphile is covalently attached to DNA.  
     
     
         14 . The complex of  claim 11  wherein the amphiphile binding agent consists of a cyclodextrin.  
     
     
         15 . A process for obtaining an expression product in vivo, comprising: 
 a. forming a complex with a cyclodextrin, an amphiphile and a polynucleotide;    b. delivering the complex to a cell in a mammal;    c. expressing the polynucleotide.    
     
     
         16 . The process of  claim 15  wherein the amphiphile binding agent is polymeric.  
     
     
         17 . The process of  claim 15  further comprising complexing the polynucleotide with a polycation.  
     
     
         18 . The process of  claim 15  further comprising associating a polyanion in step (a).  
     
     
         19 . The process of  claim 15  wherein the amphiphile consists of a polymer.  
     
     
         20 . The process of  claim 15  wherein the amphiphile consists of an interaction modifier.

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