Efficient Base Editing Strategies to Convert CAG to CAA Repeats and Diminish the Disease-Causing Mutation in Huntington's Disease
Base editing strategies employing combinations of cytosine base editors and guide RNAs can efficiently convert CAG to CAA in the huntingtin gene without generating significant indels, off-target edits, or transcriptome alterations, demonstrating their feasibility and specificity as a potential therapeutic approach for Huntington's disease.