Versatile, Robust Nanotube Delivery of siRNA and Nucleic Acids into Cells (Case 2055)

Brief Description:

The RNA interference system and its associated molecules of microRNA (miRNA) and small interfering RNA (siRNA) help control gene activity in living cells and can efficiently silence genes and gene products or otherwise alter gene expression, as a therapeutic and/or in the study of molecular pathways, e.g., signal transduction pathways/proteins.  Interference RNA has been delivered – transfected – into cells, for targeted effects, via viral and nonviral carriers and highly branched HK peptides with mixed success and safety concerns.  The lipid nature of biological membranes restricts direct intracellular delivery of potential drugs or molecular probes.  A need exists for improved and stable carriers in serum for delivery systems to be effective in a variety of cell types (e.g., primary lines, malignant, recombinant), and in vitro and in vivo scenarios.

The invention is a method for creating self-assembling, rosette nanotubes to be used as a transfection carrier-complex of one or more nucleic acids, polynucleotides, respective derivatives, mimics and inhibitors, among other molecules, into cells for therapeutic, diagnostic or scientific purposes.  Advantageously, this nanotube carrier technology does not require additional chemical modification of delivery complex components, forms nontoxic complexes without virus-related safety concerns, is very stable in water, and lacks metals associated with other known carriers.

The pharmaceutical, diagnostic and research markets will benefit from this invention, whereby, it can deliver therapeutically effective amounts of a drug or other molecule into cells to enhance or alter normal physiological functioning, or potentially perform gene therapy.  This novel carrier may be incorporated into a clinical or research transfection kit, including nanotubes, reagents and nucleic acids.

Information:

PCT application PCT/US2012/020056 is pending