Base editing: advances and therapeutic opportunities

Nat Rev Drug Discov. 2020 Dec;19(12):839-859. doi: 10.1038/s41573-020-0084-6. Epub 2020 Oct 19.

Abstract

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.

Publication types

  • Review

MeSH terms

  • CRISPR-Cas Systems*
  • Disease / genetics*
  • Gene Editing*
  • Hearing Loss / genetics
  • Hearing Loss / therapy*
  • Humans
  • Liver Diseases / genetics
  • Liver Diseases / therapy*
  • Muscular Dystrophy, Duchenne / genetics
  • Muscular Dystrophy, Duchenne / therapy*
  • Neoplasms / genetics
  • Neoplasms / therapy*
  • Polymorphism, Single Nucleotide