Mirus Bio Corporation

Roche Madison Inc.

Mirus Corporation

Hans Herweijer

Jon Asher Wolff

James E Hagstrom

Vladimir G Budker

Julia Hegge

Sean D Monahan

David L Lewis

Christine I Wooddell

Aaron G Loomis

David B Rozema

Vladimir S Trubetskoy

Paul Michael Slattum

Lori Higgs

Vladimir M Subbotin

Larry F Whitesell

Matthew R Wolff

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.

Methods for expression of transgenes

An intravenous delivery method is described that enables delivery of polynucleotides to extravascular cells of a mammalian limb. The method involves the injection of polynucleotides into a distal vein of a limb that is transiently occluded. Polynucleotide delivery is facilitated by rapid injection in sufficient volume to enable extravasation of the solution into surrounding tissue.

Intravenous delivery of polynucleotides to cells in mammalian limb

A process for delivering a polynucleotide to a cardiac tissue cell in a mammal is described, comprising introducing a composition consisting of a polynucleotide into a blood vessel and increasing permeability of the blood vessel to the polynucleotide. The polynucleotide can be a small interfering RNA or microRNA and inhibit gene expression in the cell.

Process for delivering sirna to cardiac muscle tissue

Described is a deliverable composition with low toxicity comprising an amphipathic compound, a polycation, and a siRNA. The composition may be used in the process of delivering a siRNA to an animal cell or more particularly, a mammal cell.

Compositions and processes using siRNA, amphipathic compounds and polycations

Disclosed is a system for providing in vivo delivery of molecules or complexes to extravascular mammalian cells using an intravascular administration route. The molecules or complexes are inserted in an injection solution into a mammalian vasculature. Insertion of the injection solution at an appropriate rate transiently increases the volume of extravascular fluid in the tissue thereby facilitating delivery of the molecule to the cell.

Delivery of molecules and complexes to mammalian cells in vivo

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.

Intravascular delivery of nucleic acid

We disclose compositions and processes for enhancing transposon mediated integration of a nucleic acid molecule into another target nucleic acid molecule. Integration by an integrator complex is enhanced by cationic reagents.

Enhancing intermolecular integration of nucleic acids using integrator complexes

The present invention relates to compositions and methods for transferring nucleic acids into cells in vitro and in vivo. The compositions comprise a transfection reagent and one or more detergents. In preferred embodiments, the compositions comprise delivery systems providing nucleic acid transfer complexes that transfect cells with high efficiency.

Nucleic acid transfer complexes

A process for delivering a nucleic acid to a cardiac tissue cell in a mammal is described, comprising introducing a composition consisting of a nucleic acid to a blood vessel, which subsequently delivers the nucleic acid to the cardiac tissue cell. The nucleic acid can be DNA or RNA or plasmid DNA or viral. This process is for purposes of gene therapy, and research.

Growing community of inventors

Madison, WI, United States of America

Hans Herweijer