From Chemical Design Optimization to Clinical Application: Advancing ADAR RNA Editing in Liver and CNS

• ADAR-mediated RNA editing as a versatile therapeutic approach to precisely correct disease-causing RNA mutations, modulate protein expression levels, or alter protein function to prevent or treat disease
• Optimization of editing oligonucleotides (EONs) through chemical modifications to enhance editing efficiency, specificity, and cellular uptake
• Translation of optimized EON strategies into therapeutically relevant applications addressing high unmet medical needs in the liver and CNS.

Speakers:

David Parfitt

Senior Scientist

ProQR Therapeutics