 Methods for treating viral infection using IL-28 and IL-29 |
| We claim: 1. A method of treating a viral infection comprising administering to an ... |
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| Method of manufacturing an electrohydraulic probe |
| OF THE INVENTION Referring to FIG. 1 there is shown a diagrammatical view of the bladder 2, ureter ... |
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| Releasable linkage and compositions containing same |
| Accordingly, it is an object of the invention to provide a compound where a ligand is covalently ... |
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| Solid phase dispersion of quinolone or naphthyridonecarboxylic acids |
| What is claimed is: 1. A solid phase dispersion comprising a micronized quinolinecarboxylic acid or ... |
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| Peptide, process for its preparation and pharmaceutical composition containing same |
| We claim: 1. pGlu-Pro-Pro-Gly-Gly-Ser-Lys-Val-Ile-Leu-Phe. 2. A pharmaceutical composition for cell-... |
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| Stable pharmaceutical composition and method for its production |
| It is an object of the present invention to provide a stable pharmaceutical composition wherein the ... |
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| Peptide, a method for its preparation and a pharmaceutical composition containing the peptide |
| OF THE PREFERRED EMBODIMENT The following standard abbreviations for the amino acid residues are ... |
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| Mimotopes and anti-mimotopes of human platelet glycoprotein Ib/IX |
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| Adjuvant and vaccine compositions containing monophosphoryl lipid A |
| What is claimed: 1. A lyophilized composition comprising 0.2% up to 5% by weight 3-O-desacyl-4'-... |
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Imidazole-containing cationic amphiphiles for intracellular delivery of therapeutic molecules
| Details |
Inventors: Siegel, Craig S.; Lee, Edward R.; Harris, David J.;
Assignee: Genzyme Corporation (Boston, MA)
Primary Examiner: Dees; Jose G.
Assistant Examiner: Badio; Barbara
Attorney, Agent or Firm: Finnegan, Henderson, Farabow, Garrett & Dunner, L.L.P.
Cationic amphiphiles are provided that facilitate transport of biologically active (therapeutic) molecules into cells. There are provided also therapeutic compositions prepared typically by contacting a dispersion of one or more cationic amphiphiles with the therapeutic molecules. Therapeutic molecules that can be delivered into cells according to the practice of the invention include DNA, RNA, and polypeptides. Representative uses of the therapeutic compositions of the invention include providing gene therapy, and delivery of antisense polynucleotides or biologically active polypeptides to cells. With respect to therapeutic compositions for gene therapy, the DNA is provided typically in the form of a plasmid for complexing with the cationic amphiphile. |
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DETAILED DESCRIPTION The present invention provides for cationic amphiphiles that are particularly effective to facilitate transport of biologically active molecules into cells.
Representative of such amphiphiles are those having the structure ##STR1## wherein: Z is a steroid selected from the group consisting of: ##STR2## linked by the 3-O group thereof, ##STR3## linked by the 3-O group thereof, ##STR4## linked by the 3-O group thereof, ##STR5## linked at the 3 position thereof, ##STR6## linked at the 3 position thereof, and ##STR7## linked at the 3 position thereof; R.
sup.
3 is H, or a saturated or unsaturated aliphatic group; R.
sup.
1 is an alkylamine, or a polyalkylamine; R.
sup.
4 is H, or a saturated or unsaturated aliphatic group; R.
sup.
2 is an alkylamine, or a polyalkylamine; wherein R.
sup.
1 and R.
sup.
2 are the same or different; and the ##STR8## group thereof is selected from the group consisting of: ##STR9## wherein the total number of nitrogen and carbon atoms in an R.
sup.
3 --?NH(CHR.
sup.
5).
sub.
z' !--?NH(CHR.
sup.
5).
sub.
y' !--?NH(CHR.
sup.
5).
sub.
x' ! group, or in an R.
sup.
4 --?NH(CHR.
sup.
5).
sub.
z !--?NH(CHR.
sup.
5).
sub.
y !--?NH(CHR.
sup.
5).
sub.
x ! group, is less than 40, and each of x, x', y, y', z and z' is a whole number other than 0 or 1; ##STR10## wherein the total number of nitrogen and carbon atoms in an R.
sup.
3 --?NH(CHR.
sup.
5).
sub.
y' !--?NH(CHR.
sup.
5).
sub.
x' ! group, or in an R.
sup.
4 --?NH(CHR.
sup.
5).
sub.
z !--?NH(CHR.
sup.
5).
sub.
y !--?NH(CHR.
sup.
5).
sub.
x ! group, is less than 40, and each of x, x', y, y' and z is a whole number other than 0 or 1; ##STR11## wherein the total number of nitrogen and carbon atoms in an R.
sup.
3 --?NH(CHR.
sup.
5).
sub.
x' ! group, or in an R.
sup.
4 --?NH(CHR.
sup.
5).
sub.
z !--?NH(CHR.
sup.
5).
sub.
y !--?NH(CHR.
sup.
5).
sub.
x ! group, is less than 40, and each of x, x', y and z is a whole number other than 0 or 1; ##STR12## wherein the total number of nitrogen and carbon atoms in an R.
sup.
3 --?NH(CHR.
sup.
5).
sub.
y' !--?NH(CHR.
sup.
5).
sub.
x' ! group, or in an R.
sup
|
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Gene Therapy 
