Activation of Therapeutic Functionalities with Chimeric RNA/DNA Nanoparticles for Treatment of Cancer, Viruses and Other Diseases

A new strategy based on RNA/DNA hybrid nanoparticles, which can be generally used for triggering multiple functionalities inside diseased cells is presented. Individually, each of the hybrids is functionally inactive and functional representation can only be activated by the re-association of at least two cognate hybrids simultaneously present in the same cell. Overall, this novel approach allows (i) the triggered release of therapeutic siRNAs or miRNAs inside the diseased cells, (ii) activation of other split functionalities (e.g. FRET, different aptamers, rybozymes, split proteins) intracellularly, (iii) higher control over targeting specificity (e.g. if two hybrids are decorated with two different tissue specific recognition moieties), (iv) biosensing and tracking of the delivery and re-association of these hybrids in real-time inside cells, (v) increasing the number of functionalities by introducing a branched hybrid structure, (vi) introduction of additional functionalities without direct interference of siRNA processivity, (vii) increasing the retention time in biological fluids by fine-tuning chemical stability through substituting the DNA strands with chemical analogs (e.g. LNA, PNA, etc), (viii) conditional release of all functionalities.IC: NCINIH Ref. No.: E-039-2012/0TAB No: TAB-2496Advantages: Novel way for multiple functionality delivery and activationEnhanced chemical stability and pharmacokinetics due to the average size of nanoparticles ex...
Source: NIH OTT Licensing Opportunities - Category: Research Authors: Source Type: research