Genzyme Corporation

S Catherine Hubbard

Seng H Cheng

Craig S Siegel

David J Harris

John Marshall

Edward R Lee

Ronald K Scheule

Simon J Eastman

Chau-Dung Chang

Rebecca G Bagley

H Michael Shepard

Richard J Gregory

Nelson S Yew

Daniel C Maneval

Duane E Johnson

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.

Organ-specific targeting of cationic amphiphile / DNA complexes for gene therapy

Cationic amphiphile/DNA complexes

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Belmont, MA, United States of America

S Catherine Hubbard