RNAfix: Therapeutic RNA Editing with Engineered Guide RNAs and Endogenous ADAR

Time: 9:40 am
day: Day One


  • Understanding endogenous ADAR enzyme can be harnessed to edit adenosines in RNA and treat disease. A massively parallel screen of guide RNAs (gRNAs) identifies designs that enable highly efficient and specific editing of clinically
    relevant targets
  • Divulging that machine learning analysis of deep gRNA screening datasets allows computational generation of gRNAs with improved performance relative to any designs in the original screen
  • Discussing identified gRNAs can be further optimized and genetically delivered with AAV to enable therapeutic levels of editing in multiple cell types