Current gene therapy

Progress and Challenges in Using Adenine Base Editors for Biology and Medicine

Updated

Abstract

Adenine base editors (ABEs) enable precise A·T to G·C conversions in the genome.

  • ABE technology has advanced significantly in applications for genetic diseases, including spinal muscular atrophy and mitochondrial disorders.
  • Major therapeutic advances using ABEs are noted in conditions like hyperlipidemia.
  • Potential uses of ABEs in agriculture include enhancing genetic traits in crops such as grains and fruits.
  • AE performance has been systematically compared to other base editing technologies, revealing both strengths and limitations.
  • Biosecurity risks, including off-target effects and genomic instability, are critical concerns associated with ABE applications.
  • Safety issues are identified as a significant barrier to the clinical use of ABEs in precision medicine.

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