Unlocking the Potential of RNA Biology and Therapeutics

DNA’s second-in-command could help treat a variety of diseases.

It’s common knowledge that our DNA plays an essential role in health and illness, but considerably less is known about the impact of its companion molecule, ribonucleic acid (RNA). Once thought to simply relay DNA’s instructions to the cell’s protein-creating machinery, we now know that RNA comes in several varieties that have tremendous influence over how genes behave. As a result, RNA presents an extremely promising avenue for the treatment of many conditions.

The IRP’s strong foundation in RNA biology research has allowed our investigators to make numerous important discoveries about the molecule itself and how it can be leveraged to fight disease. Much of this work has focused on RNA interference (RNAi), a process by which small strands of RNA called small interfering RNAs (siRNAs) reduce or eliminate the activity of a gene. So far, IRP scientists have capitalized on RNAi to develop potential therapeutic approaches for neurodegenerative diseases like Parkinson’s disease, hemorrhagic fever viruses, and even certain cancers. Our researchers are also leading the way on creating methods to deliver RNA-based treatments directly to tumors.

Our increasing understanding of RNA, combined with the emergence of new, cutting-edge technologies for studying it, is now hastening the clinical revolution promised by RNA-based therapies. In particular, the Trans-NIH RNAi Screening Facility has proven to be an incredible boon to IRP researchers by allowing them to rapidly test how the gene-silencing effects of thousands of different siRNA molecules affect normal and diseased cells. Facility staff help investigators with all aspects of project planning and execution, including assisting with the setup for RNAi screens, providing access to numerous commercially available collections of siRNA molecules, and offering tools for storing, tracking, and analyzing data. The RNAi program also conducts research of its own to advance the science of RNAi screening, such as efforts to reduce the rate of false-positives.

The powerful tools and tremendous expertise of the IRP community continue to advance the fields of RNA biology and therapeutics. Our investigators are moving towards RNA-based treatments by:

• Mapping the collection of RNA molecules — known as the RNAome — in normal and malfunctioning cells
• Pursuing improvements to RNAi screening methods
• Investigating the processes by which RNA is created and modified within cells
• Studying how RNA molecules interact with DNA, proteins, and one another
• Elucidating the structure of RNA
• Establishing the role of RNA in disease and exploring RNA-based and RNA-targeted therapies

Explore these pages for more information about the past, present, and future of IRP research on RNA biology and therapeutics:

• RNA Biology Scientific Focus Area
• RNAi Screening Facility

Check out all 12 of the domains in which we are Accelerating Science to learn about how IRP scientists are tackling important biomedical challenges.