The National Cancer Institute's Protein Expression Laboratory seeks parties interested in licensing the novel delivery of RNA to mammalian cells using virus-like particles.

Current methods of delivering proteins or RNA to mammalian cells are limited by a lack of target specificity and toxicity, among other shortcomings.  NCI researchers have created novel virus-like particles (VLPs) that are capable of binding to and replicating within a target mammalian cell, including human cells.  The claimed VLPs are safer than viral delivery because they are incapable of re-infecting target cells.  The present VLPs can optionally comprise inhibitory recombinant polynucleotides, such as microRNA, antisense RNA or small hairpin RNA, to down regulate or turn off expression of a particular gene within the target cell.  Alternatively, recombinant polynucleotides packaged within VLPs can comprise a gene encoding a therapeutic protein so as to enable expression of that protein within the target cell.   Specifically, VLPs of the invention are composed of an alphavirus replicon that contains a recombinant polynucleotide, a retroviral gag protein, and a fusogenic envelope glycoprotein.

 While the claimed VLPs have a variety of applications, therapeutic uses of the VLPs include directing antibody synthesis and converting cancer cells into antigen presenting cells. Additional applications include using VLPs to induce fast (approx. 3-4 hrs) and high levels of protein production in mammalian cells.

Competitive Advantages:

• Obviates the need to use expensive antigen purification for proteins or antigens produced inside target cells
• High level (~million copies per cell) of RNA production/synthesis within target cell
• Fast expression (approx. 3-4 hrs compared to 1-2 days) following VLP introduction into target cells

Commercial Applications:

• Delivery of microRNA and small hairpin RNA to reduce expression of targeted genes in a human cell
• Delivery of coding RNA for robust expression in mammalian systems
• Direct antibody production by in vivo injection of replicons (no antigen purification)