Absolute quantification of RNA editing with digital PCR

RNA editing is a crucial, naturally occurring process that allows cells to change the information in RNA after it has been transcribed. To harness this mechanism, ProQR has introduced the innovative Axiomer® technology, which includes synthetic RNA strands called editing oligonucleotides (EONs). EONs are designed to mimic the double-stranded structure that Adenosine Deaminse Acting on RNA (ADAR), an A-to-I editing enzyme, recognizes.  EONs effectively attract ADAR to specific locations within the RNA molecule for precise A-to-I edits. This technology offers a new way to treat diseases, allowing for the correction of RNA carrying disease-causing mutations or the modification of proteins to prevent or treat various conditions. It utilizes the body's own RNA editing machinery, eliminating the need to introduce foreign proteins, and simplifying manufacturing and delivery processes compared to gene therapy or gene editing. Additionally, the transient nature of RNA editing reduces the risk of long-lasting side effects.

In this webinar, we explain how Axiomer® technology works and discuss the use of digital polymerase chain reaction (dPCR) to measure the extent of A-to-I editing. Digital PCR provides a highly detailed, quantitative method for measuring the level of A-to-I editing, enhancing our understanding of the technology's effectiveness and its potential to address a range of diseases. The process involves identifying target RNA sites amenable to A-to-I edits, designing, and delivering EONs, ADAR-mediated editing, and producing corrected or modified proteins, thereby altering the course of diseases. Digital PCR is a valuable companion technique, facilitating precise measurement of RNA editing within this framework.

About the speaker
Dr. Kristin Bell, Senior Scientist
Northern Biomolecular Services
Dr. Kristin Bell is currently a Study Director at Northern Biomolecular Services, where she oversees preclinical and clinical bioanalysis to support GLP- and non-GLP-compliant safety and efficacy studies. Prior to her involvement in the initiation and startup of Northern Biomolecular Services in 2019, she worked as a laboratory scientist to develop laboratory assays primarily for branch DNA and (RT)qPCR analysis.
Date of recording:Tuesday, November 14, 2023
Duration:60 minutes
Pharma / Biopharma
Drug Safety / Pharmacology
Digital PCR