Abstract: A low toxicity, highly efficient transfection composition is described with an amphipathic compound containing at least one imidazole. The composition may be used in the process of transfecting nucleic acids into an animal cell.

Abstract: Chelator containing compounds are utilized in the delivery of molecules and polymers to animal cells. At least one chelator such as crown ether is attached to a polymer and then associated with another polymer. An ion is then added to the mixture thereby forming condensed polymer. In condensed form and in complex with the chelator, polymer can be delivered to a cell.

Abstract: Disclosed is expression of zeta negative and zeta positive nucleic acids or nucleic acid complexes using a dystrophin gene in a process for providing nucleic acid expression in a striated (skeletal or cardiac) muscle cell for the purpose of providing a change to the endogenous properties of the cell for cells affected by muscular dystrophy.

Abstract: Described is a process for intravascular delivery of a polynucleotide to an extravascular cell of a mammal to inhibit gene expression. A polynucleotide containing sequence that is similar to a sequence in the gene to be expressed is made and inserted into a vessel in the mammal. The polynucleotide is delivered to a cell wherein expression of the gene is inhibited.

Abstract: Processes are described for obtaining high levels of gene expression in primates after injection of nucleic acid to the liver via the lumen of the hepatic vein. The described process results in high level of gene expression with transient increases in liver enzymes.

Abstract: An polyampholyte is utilized in a condensed polynucleotide complex for purposes of nucleic acid delivery to a cell. The complex can be formed with an appropriate amount of positive and/or negative charge such that the resulting complex can be delivered to the extravascular space and may be further delivered to a cell.

Abstract: We describe pH-sensitive endosomolytic polymers, delivery particles containing pH-sensitive endosomolytic polymers. The described particles are capable of delivering polynucleotides to cells from the peripheral circulation with subsequent release from endosomes. The endosomolytic polymers are inactive outside the cell but disrupt membranes upon exposure to an acidified endosomal compartment.

Abstract: Methods are described for modifying nucleic acids to facilitate delivery of the nucleic acids to cells. Compounds which interact with of modify nucleic acids are interacted with the nucleic acids within reverse micelles.

Abstract: Processes are described for obtaining high levels of gene expression in primates after injection of nucleic acid to the liver via the lumen of the hepatic vein. The described process results in high level of gene expression with transient increases in liver enzymes.

Abstract: Compounds and methods are provided for a single-pot covalent attachment of a label to nucleic acids comprising forming a covalently attachable labeling reagent for alkylating the molecule. Then, combining the covalently attachable labeling reagent with a mixture containing the molecule, under conditions wherein the labeling reagent has reactivity with the molecule thereby forming a covalent bond.

Abstract: A method of forming polymers in the presence of nucleic acid using template polymerization. These methods can be used for the delivery of nucleic acids, for condensing the nucleic acid, for forming nucleic acid binding polymers, for forming supramolecular complexes containing nucleic acid and polymer, and for forming an interpolyelectrolyte complex.

Abstract: An process for the reversible modification of an amine-containing compound is described. Modification of the compound can be used to facilitate delivery of molecules to cells in vitro and in vivo or to alter interactions or activities the compounds. The described modifiers can also be utilized as cross-linkers.

Abstract: Disclosed is a process for providing for expression of an exogenous nucleic acid in an extravascular parenchymal cell of a mammal. The nucleic acid is inserted into a vessel of a mammal and the permeability of the vessel is increased. Increasing permeability of the vessel allows delivery of the nucleic acid to an extravascular parenchymal cell.

Abstract: A process is provided to deliver polynucleotide-based gene expression inhibitors to cells in a mammal in vivo for the purpose of inhibiting gene expression in the cells. Inhibition is sequence-specific and relies on sequence similarity to of the polynucleotide-based gene expression inhibitor and the target nucleic acid molecule. Delivery of the polynucleotide-based gene expression inhibitor can enhance the efficacy of co-delivered small molecule drugs.

Abstract: Polyampholyte are able to condense nucleic acid to form small complexes which can be utilized in the delivery of nucleic acid to mammalian cells. The polyampholytes can be formed prior to interaction with nucleic acid or they can be formed in the presence of nucleic acid. Stabilized polycation/nucleic acid complexes can be modified to reduce the positive charge of the polycation and add targeting ligands without destabilizing the complex. The resultant particles retain their small size and are more effective in delivery of nucleic acid to cells in vivo.

Abstract: A method of forming polymers in the presence of nucleic acid using template polymerization. Also, a method of having the polymerization occur in heterophase systems. These methods can be used for the delivery of nucleic acids, for condensing the nucleic acid, for forming nucleic acid binding polymers, for forming supramolecular complexes containing nucleic acid and polymer, and for forming an interpolyelectrolyte complex.

Abstract: A process and compound wherein nucleic acids can be modified with a host of molecules and maintain their ability to be expressed. A modifying chemical attachment of polyions to polynucleotides can be used to facilitate the change of tertiary structure of the nucleic acid and in some cases condensation of nucleic acids into smaller, charged particles useful in delivering the nucleic acid to a cell.

Abstract: The invention provides for polycations for condensation and delivery of polynucleotides to cells. Processes for forming the polycations by the polymerization of formamide monomers is also described.

Abstract: A process is provided to deliver small interfering RNA to cells in vivo for the purpose of inhibiting gene expression in that cell. The small interfering RNA is less than 50 base-pairs in length. This process is practiced on post-embryonic animals. Inhibition is sequence-specific and relies on sequence identity of the small interfering RNA and the target nucleic acid molecule.

Abstract: An process for the reversible modification of membrane interaction of a compound is described. Modification of membrane interaction can be used to facilitate delivery of molecules to cells in vitro and in vivo. The described modifiers, which are used to reversibly inactivate the membrane active compounds, can also be utilized as cross-linkers or to reverse the charge of a molecule.