Base editing: advances and therapeutic opportunities

Base editing — the introduction of single-nucleotide variants (SNVs) into DNA or RNA in living cells — is one of the most recent advances in the field of genome editing. As around half of known pathogenic genetic variants are due to SNVs, base editing holds great potential for the treatment of numerous genetic diseases, through either temporary RNA or permanent DNA base alterations. Recent advances in the specificity, efficiency, precision and delivery of DNA and RNA base editors are revealing exciting therapeutic opportunities for these technologies. We expect the correction of single point mutations will be a major focus of future precision medicine. Base editing is emerging as a potential approach to treat genetic diseases through the introduction of single-nucleotide alterations in DNA and RNA. Here, Porto et al. provide an overview of DNA and RNA base editing, discuss recent advances in the development of these technologies and highlight promising therapeutic applications.