RNA Editing: A New Approach to Precision Medicine

• RNA editing can make temporary changes to a person’s genome, allowing doctors to stop therapy if a problem arises, mitigating long-term risk.
• At least 11 companies are developing RNA editing methods for various diseases, attracting interest from large pharmaceutical firms like Eli Lilly, Roche, and Novo Nordisk.
• Wave Life Sciences, a biotechnology company in Massachusetts, was the first to treat a genetic condition by editing RNA at the clinical level.
• RNA editing allows scientists to fix mistakes in the mRNA after the cell has synthesised it but before the cell reads it to make the proteins.
• Wave Life Sciences used RNA editing to treat -1 antitrypsin deficiency (AATD), an inherited disorder affecting the liver and lungs.
• Other companies using ADAR enzymes for RNA editing include Korro Bio, ProQr Therapeutics, and Shape Therapeutics.
• RNA editing has advantages over DNA editing, including safety and flexibility, and relies on ADAR enzymes, which are already present in the human body, thus presenting a lower risk of allergic reactions.
• Challenges in RNA editing include the specificity of ADARs in RNA editing, the transient nature of RNA editing, and the limited carrying capacity of current methods to deliver the gRNA-ADAR complex.