Children's Medical Center Corporation

Immune Disease Institute, Inc.

Industry-University Cooperation Foundation Hanyang University

Premlata Shankar

Manjunath Narasimhaswamy

Priti Kumar

Sang-Kyung Lee

Judy Lieberman

Erwei Song

Haoquan Wu

Hong-Seok Ban

Manju Swamy

The present invention provides compositions and methods useful for delivering agents to target cells or tissues, for example nerve cells and other cells in the central nervous system. The compositions and methods are useful for delivering agents across the blood-brain barrier. The present invention also provides methods of using the compositions provided by the present invention to deliver agents, for example therapeutic agents for the treatment of neurologically related disorders.

Peptides for assisting delivery across the blood brain barrier

The present invention provides a method of delivering RNA interference molecules to a cell or a cell in a subject, which comprises contacting the cell with a protein-double stranded RNA complex, the complex comprising the double stranded RNA segment containing a double stranded RNA of interest and a protein, the protein comprising (1) a targeting moiety, which will specifically bind to a site on a target cell, and (2) a binding moiety linked thereto, which will bind to the double stranded RNA, wherein the double stranded RNA segment is delivered to a cell and effects RNA interference of the target RNA in the cell.

Targeted delivery of siRNA

Method for delivery across the blood brain barrier

The present invention is based, at least in part, on the discovery of compositions and methods for the treatment and prevention of infectious diseases or disorders, e.g., HIV infection, AIDS, and AIDS-related diseases. In particular, the present invention pertains to methods of modulating cellular gene expression or protein activity, e.g., CCR5, gene expression or protein activity and/or gene expression or protein activity of a gene or sequence of an infectious agent, in order to treat or prevent infectious diseases or disorders, HIV infection, AIDS, or an AIDS-related disease or disorder. In one embodiment the combination of an RNA interfering agent targeting a cellular gene in combination with an RNA interfering agent targeting a gene or sequence of an infectious agent results in prolonged prevention of infection by an infectious agent. The present invention is based on the identification of novel RNA interference agents, e.g., siRNA molecules, which target cellular genes, e.g., chemokine receptors, e.g., the CCR5 gene, and result in inhibition of target gene expression on target gene expressing cells, thereby inhibiting entry of infectious agents, e,g., HIV infection into target cells, prevention infection, and/or suppressing replication in established infection.

Inhibition of gene expression using RNA interfering agents

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El Paso, TX, United States of America

Premlata Shankar