Article

DCLRE1A orchestrates CAG repeat contraction following Cas12a-induced DNA breaks

2025-11-18

Abstract excerpt

<h4>ABSTRACT</h4> Despite their well-defined genetic background, repeat expansion diseases (REDs) still represent an unmet medical need, with no causative therapy offered to patients. The strategy of repeat shortening using genome editing tools is very attractive because a single intervention can result in permanent repair of the disease-causing mutation. However, a limited understanding of DNA repair mechanisms...

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Literature Corpus work: 1c4cefe2-2992-537b-ae83-5b659b31a41f
DOI: 10.1101/2025.11.18.689026

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