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亨廷顿病（HD）是一种棘手的遗传性神经退行性疾病。研究人员围绕 HD 中体细胞重复不稳定性展开研究，发现错配修复（MMR）酶加剧 HTT 基因中 CAG 三联体重复不稳定。多家公司据此开发相关疗法，有望为 HD 治疗带来新突破。