The present invention relates to piperazine based amphilic cationic lipids useful for gene therapy, transfection, and introducing immunogenic compounds for the purpose of vaccination. The disclosed compounds have lipophilic moieties linked to the ring nitrogens. In addition, at least one of the ring nitrogens is quatranized and linked to a hydrocarbon having at least one heteroatom.
A novel nucleoside analog is disclosed which comprises a piperazine ring in the place of the ring ribose or deoxyribose sugar. Monomers utilizing a broad variety of nucleobases are disclosed, as well as oligomers comprising the monomers disclosed herein linked by a variety of linkages, including amide, phosphonamide, and sulfonamide linkages. A method of synthesizing the nucleoside analogs is also disclosed.
The present invention relates to piperazine based amphilic cationic lipids useful for gene therapy, transfection, and introducing immunogenic compounds for the purpose of vaccination. The disclosed compounds have lipophilic moieties linked to the ring nitrogens. In addition, at least one of the ring nitrogens is quatranized and linked to a hydrocarbon having at least one heteroatom.
The present invention discloses highly packed polycationic ammonium, sulfonium and phosphonium lipid compounds useful for making lipid aggregates for delivery of macromolecules and other compounds into cells. They are especially useful for the DNA-dependent transformation of cells. Methods for their preparation and use as intracellular delivery agents are also disclosed.
The present invention discloses highly packed polycationic ammonium, sulfonium and phosphonium lipid compounds useful for making lipid aggregates for delivery of macromolecules and other compounds into cells. They are especially useful for the DNA-dependent transformation of cells. Methods for their preparation and use as intracellular delivery agents are also disclosed.
A composition is provided comprising a novel cationic lipid compound having hydrophobic tails and two quaternary ammonium headgroups bridged by a linker. The composition is useful as a cytofectin for facilitating delivery and transfection of biologically active agents, particularly anionic bioactive agents such as DNA, into cells. The composition is useful also as an adjuvant for enhancing the humoral immune response of a vertebrate to an immunogen, especially an immunogen encoded by a polynucleotide-based vaccine. In certain preferred embodiments, the cationic lipid compound is a dimer containing quaternary ammonium headgroups bridged by a linker having DNA and/or cell receptor binding affinity, such as a polypeptide or polyamine. Also disclosed is an immunogenic composition comprising an immunogen and the composition of the present invention.
