US2002133126A1PendingUtilityA1

Transfection system, its preparation and use in somatic gene therapy

Priority: Jul 11, 1997Filed: Jan 12, 2001Published: Sep 19, 2002
Est. expiryJul 11, 2017(expired)· nominal 20-yr term from priority
Inventors:Jurgen Schrader
C12N 2750/14143C12N 15/86A61K 48/00C12N 9/0075
51
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Claims

Abstract

The present invention relates to a transfection system comprising one or more Infiltrator catheters, one or more nucleic acids, and, where appropriate, suitable ancillary substances and/or additives, and to its preparation and use in somatic gene therapy.

Claims

exact text as granted — not AI-modified
We claim:  
     
         1 . A transfection system comprising 
 (a) one or more Infiltrator catheters;    (b) one or more nucleic acids in nonviral form; and, where appropriate,    (c) suitable ancillary substances and/or additives.    
     
     
         2 . A transfection system as claimed in  claim 1 , wherein the Infiltrator catheter comprises one or more injector ports with a height of about 100-500 μm.  
     
     
         3 . A transfection system as claimed in  claim 1 , wherein the Infiltrator catheter comprises about 5×7 injector ports.  
     
     
         4 . A transfection system as claimed in  claim 1 , wherein the Infiltrator catheter is a catheter with more than one lumen.  
     
     
         5 . A transfection system as claimed in  claim 1 , wherein the Infiltrator catheter comprises a central tubular shaft, an inflatable balloon which envelops the shaft at a suitable point, and a tubular sleeve which comprises one or more injector ports at the point where the inflatable balloon is.  
     
     
         6 . A transfection system as claimed in  claim 1 , wherein the transfection system additionally comprises a guide wire.  
     
     
         7 . A transfection system as claimed in  claim 1 , wherein the nucleic acid is selected from the group consisting of a nucleic acid in naked form and a nucleic acid together with other nonviral components.  
     
     
         8 . A transfection system as claimed in  claim 1 , wherein the nucleic acid is selected from the group consisting of a single- or double-stranded DNA or RNA, a plasmid DNA and an antisense oligonucleotide.  
     
     
         9 . A transfection system as claimed in  claim 1 , wherein the nucleic acid is present in the form of a nucleic acid/liposome complex.  
     
     
         10 . A transfection system as claimed in  claim 9 , wherein the nucleic acid/liposome complex also comprises one or more nucleic acid binding proteins.  
     
     
         11 . A transfection system as claimed in  claim 1 , wherein the nucleic acid codes for a therapeutically effective gene product.  
     
     
         12 . A transfection system as claimed in  claim 11 , wherein the therapeutically effective gene product is a nitric-oxide synthase.  
     
     
         13 . A transfection system as claimed in  claim 11 , wherein the nucleic acid coding for a therapeutically effective gene product comprises one or more noncoding sequences and/or a polyA sequence.  
     
     
         14 . A process for producing a transfection system comprising 
 (a) one or more Infiltrator catheters;    (b) one or more nucleic acids in nonviral form; and, where appropriate,    (c) suitable ancillary substances and/or additives, wherein one or more Infiltrator catheters, one or more nucleic acids in nonviral form and, where appropriate, suitable ancillary substances and/or additives and, where appropriate, a guide wire are combined.    
     
     
         15 . Method of using of a transfection system comprising 
 (a) one or more Infiltrator catheters;    (b) one or more nucleic acids in nonviral form; and, where appropriate,    (c) suitable ancillary substances and/or additives, for somatic gene therapy.    
     
     
         16 . Method as claimed in  claim 15  for treating vascular disorders, genetically related disorders and/or disorders which can be treated by gene transfer, including prevention thereof.  
     
     
         17 . Method of using an Infiltrator catheter for producing a transfection system comprising 
 (a) one or more Infiltrator catheters;    (b) one or more nucleic acids in nonviral form; and, where appropriate,    (c) suitable ancillary substances and/or additives.

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