摘要
桥本甲状腺炎(HT)是一种常见的自身免疫性内分泌疾病。它通常与其他众所周知的疾病(如1型胰岛素依赖型糖尿病)一起出现在儿科年龄组。这种疾病的主要特征是免疫介导的对甲状腺的攻击,导致甲状腺组织和细胞的破坏。鉴于HT经常影响家庭成员,人们普遍认为个体在遗传上易患这种疾病。HT患者还显示出几种不同癌症的风险显著增加,证明了开发管理和治疗HT疗法的迫切需要。基因编辑在分子生物学领域取得了一些进展,并已成为纠正几种自身免疫性疾病的有前途的方法。目前,CRISPR/Cas是一种基于核酸酶的编辑技术,被宣传为治疗多种遗传疾病和癌症的有前途的工具。然而,目前通过CRISPR/Cas技术对自身免疫性疾病的管理和治疗进行的研究非常有限。本综述提供了与桥本甲状腺炎相关的潜在候选基因的描述,并且只有少数动物和人类模型通过CRISPR/Cas基因编辑技术生成。通过CRISPR/Cas基因编辑技术靶向候选基因生成自身免疫性甲状腺炎小鼠模型,将为我们更深入地了解HT的病理生理,并进一步为基因编辑对HT的免疫调节铺平道路。
关键词: 桥本甲状腺炎,CRISPR/Cas, TSH激素,Cas9,基因组编辑,免疫调节。
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