Article

Compact adenine base editors to enable therapeutic rescue of Duchenne muscular dystrophy

2026-05-12

Abstract excerpt

<h4>ABSTRACT</h4> Adenine base editors (ABEs) have emerged as a powerful gene-editing technology enabling precise and programmable adenine-to-guanine substitutions across the genome. However, their translation into in vivo therapeutics is limited by delivery challenges, as their size exceeds the packaging capacity of adeno-associated virus (AAV). Here, we report the discovery, structural characterization, and eng...

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Literature Corpus work: 38d7afea-a49c-5092-9992-19113a6aed69
DOI: 10.64898/2026.05.08.723843

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