肝脏疾病的现代治疗方法

肝脏 ›› 2022, Vol. 27 ›› Issue (9): 950-952.

肝脏疾病的现代治疗方法

贾昊宇, 杨长青

200065 上海同济大学附属同济医院消化科,同济大学医学院消化疾病研究所

收稿日期:2022-08-25 出版日期:2022-09-30 发布日期:2022-10-27
通讯作者: 杨长青,Email: cqyang@tongji.edu.cn
基金资助:
国家自然科学基金项目 (81670571,81820108006);上海市临床重点专科建设项目( SHSLCZDZK06801);上海申康三年行动计划重大临床研究项目(SHDC2020CR2030B)

Received:2022-08-25 Online:2022-09-30 Published:2022-10-27

摘要/Abstract

摘要： 肝脏是生产和加工的关键器官,对人类健康至关重要。肝功能障碍可导致全身性和肝脏特异性疾病。为治疗这些疾病,开发出了基于基因添加/编辑或基因沉默的具有高度肝脏靶向性的遗传学方法。对血友病以及神经肌肉疾病患者使用的基因添加/编辑疗法已初显成效,并衍生出大量肝脏相关疾病的临床试验。然而,治疗后的免疫应答和长期输入外源载体表达的稳定性仍然是重要的挑战。利用基因编辑和mRNA技术已经产生了首个人类概念验证疗法和疫苗,但在治疗肝脏相关疾病领域仍有待突破。

关键词: mRNA疗法, 基因添加, 基因编辑, 基因沉默, 肝脏靶向治疗

贾昊宇, 杨长青. 肝脏疾病的现代治疗方法[J]. 肝脏, 2022, 27(9): 950-952.

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