Researcher South Korea

Seoul National University Base Editing Team

Seoul National University College of Medicine

Published breakthrough on DdCBE (dual-deaminase base editor) for mitochondrial DNA correction in Nature Communications (Nov 2025). Represents the most advanced preclinical approach to directly correcting LHON mutations rather than replacing gene expression.

Research focus

base editingDdCBEmtDNA correctionpreclinical

Public profiles

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Relevant project findings

The SNU team’s base editing approach is fundamentally different from existing AAV2-based gene therapies — it corrects the underlying mtDNA mutation rather than introducing a functional copy of the gene. If it advances to clinical trials, it could be transformative. Currently preclinical but positioned for first-in-human trials within 2-4 years.

Project outreach status

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