Drug – bio-affecting and body treating compositions – Designated organic active ingredient containing – Carbohydrate doai
Patent
1996-02-01
1998-07-21
Chambers, Jasemine C.
Drug, bio-affecting and body treating compositions
Designated organic active ingredient containing
Carbohydrate doai
424450, 252357, 536 231, 552544, 935 52, 935 54, A61K 9127, A61K 4800, B01F 1716, C12N 1500
Patent
active
057835659
ABSTRACT:
Novel cationic amphiphiles are provided that facilitate transport of biologically active (therapeutic) molecules into cells. The amphiphiles contain lipophilic groups derived from steroids, from mono or dialkylamines, or from alkyl or acyl groups; and cationic groups, protonatable at physiological pH, derived from amines, alkylamines or polyalkylamines. 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.
Novel and highly effective plasmid constructs are also disclosed, including those that are particularly effective at providing gene therapy for clinical conditions complicated by inflammation. Additionally, targeting of organs for gene therapy by intravenous administration of therapeutic compositions is described.
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Cheng Seng H.
Eastman Simon J.
Harris David J.
Lee Edward R.
Marshall John
Chambers Jasemine C.
Genzyme Corporation
Razzaque Abdur
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