Invention Description
While recent advances have made RNA therapeutics and vaccines more mainstream, they still suffer from drawbacks including high-dosing requirements and the inability for conditional and targeted expression. Further, they can experience off-target gene expression and related side effects as well as challenges in tissue- or cell-specific delivery of RNA therapeutics.
Researchers at the Biodesign Institute of Arizona State University have developed an innovative platform so that mRNA can be switched on only in the presence of a specific trigger, thus enabling conditional gene expression. This technology is appropriate for both vaccine delivery as well as gene therapies in target cells with known transcriptomic abnormalities. It can accommodate delivery of neoantigens, multiple immunological epitopes as well as full length coding sequences. Because this system allows efficient gene delivery and precise activation only when triggered, it overcomes the challenges of high dosing and non-specific expression plaguing RNA vaccines and gene therapies.
This platform provides a stable, compact gene delivery system with a built-in ON switch that enables targeted gene expression for improved RNA vaccines and therapeutics.
Potential Applications
- Next-generation RNA vaccines with improved safety and efficacy
- Targeted gene therapies for specific tissues or cell types
- Precision medicine platforms requiring conditional gene expression
- Biopharmaceutical development for controlled gene activation
Benefits and Advantages
- Stable and compact gene delivery system
- Built-in ON switch for conditional, cell-specific gene expression
- Reduced off-target effects and enhanced safety profile
- Efficient loading and manipulation of mRNA for controlled activation
- Improves precision in gene therapy and RNA vaccine delivery
- Allows for precise stochiometric co-delivery of multiple different mRNAs
- Enhanced stability over single stranded RNA
- Higher packing density in some delivery modes
For more information about the inventor(s) and their research, please see