Revolutionizing genetic disease treatment: Recent technological advances in base editing

Treating human genetic diseases has posed a significant and longstanding challenge to the medical community with traditional approaches often limited by their targeting scope, precision, and safety. In recent years, the development of Base Editors (BEs) has revolutionized the genome editing field, offering a precise and effective therapeutic option for correcting single nucleotide mutations responsible for more than half of human genetic diseases without causing unintended DNA damage. Since their invention in 2016, BEs have been successfully used in both research and clinical settings to treat many human genetic diseases, including sickle cell disease and amyotrophic lateral sclerosis. This review summarizes the most impactful developments in BE technology between 2020 and 2023, particularly emphasizing the methodology used to enhance this revolutionary tool. By offering a comprehensive overview of the latest advances in BE technology, we hope to provide a framework to accelerate future innovations in this field.