摘要:
The present invention relates to compositions and methods for increasing long term expression in vitro and in vivo, comprising sequences and gene expression cassettes that increase expression of genes to which they are operably linked.
摘要:
The present invention is directed compositions for targeted delivery of RNA interference (RNAi) polynucleotides to hepatocytes in vivo. Targeted RNAi polynucleotides are administered together with co-targeted delivery polymers. Delivery polymers provide membrane penetration function for movement of the RNAi polynucleotides from outside the cell to inside the cell. Reversible modification provides physiological responsiveness to the delivery polymers.
摘要:
Described is a process for delivering a biologically active compound to a cell by reversibly linking the compound to a membrane active polymer. In particular, polymer-polynucleotide conjugates are described. Methods for reversibly modifying the polymers to decrease cellular toxicity and improve efficacy are provided.
摘要:
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.
摘要:
Described are drug formulations that increase regional delivery of the drugs to cells. Methods for reversibly increasing the hydrophobicity of a drug through hydrolytically labile attachment of a hydrophobic moiety and methods for delivering the modified drug to cells are described. Hydrophobic modification increases drug delivery, while lability minimizes entry of the drug into non-target cells.
摘要:
We describe compounds that bind to and are internalized by hepatocytes. Association of these compounds to other molecules or complexes can be used to target the molecules or complexes to hepatocytes in vivo or in vitro.
摘要:
Cyclic amidinium containing compounds and their methods of preparation are described. Compositions containing these compounds facilitate delivery of biologically active polymers to cells in vitro and in vivo.
摘要:
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.
摘要:
The present invention is directed to membrane active polymers useful for cellular delivery of compounds. Described are polyconjugates systems that incorporate targeting, anti-opsonization, anti-aggregation, and transfection activities into small biocompatible in vivo delivery conjugates. The use of reversible modification provides for physiologically responsive activity modulation.
摘要:
We describe a class of polymaleic anhydride polymers capable of disrupting cell membranes. Co-delivery of these polymers with biologically active compounds increases cellular cytoplasmic delivery of the compounds.