Gene therapy research has yielded FDA-approved treatments for an array of diseases. However, challenges facing nucleic-acid based therapeutics include non-specific delivery and degradation of the nanoparticles. NCI investigators have developed a solution to address these challenges in their novel nucleic-based therapy based on the conditional activation strategy.
The inducible activation nucleic acid hybrid switch overcomes the drawbacks of current technologies through its unique design. The implementation of nucleic acid logic elements in these constructs circumvents off-target effects. The functional oligonucleotide constructs would only be generated and activated in environments denoted by the presence or absence of a specific cognate RNA trigger, ensuring context-sensitive function. The systems were also optimized to be resistant to nuclease degradation yet inexpensive for commercial production. Furthermore, this collection of logic-based systems can accommodate different trigger/target sequence pairs, enhancing its diversity of application, and serves as a novel paradigm for conditionally regulated therapeutics against cancer, genetic disorders, or infectious diseases. The NCI is looking for innovative companies interested in co-developing and/or licensing this technology.