CRISPR是一种基因编辑工具，改编自细菌中天然存在的防御系统。这项技术正在革新研究基因在肝脏疾病中的功能驱动过程，尤其是通过基因筛选和动物基因敲除/敲入模型的推动。该技术正在用于肝脏疾病模型中，以确定哪些基因对肝脏病理非常重要，尤其是在遗传性肝病、肝炎和癌症的起始和发展中。它在直接编辑DNA方面在治疗人类疾病方面具有巨大的潜力。它可以针对表达失调蛋白质的基因功能进行无效化，例如阻断转甲状腺综合征作为淀粉样变病的治疗，或者矫正基因缺陷，例如恢复正常的肝酶富马酰乙酰乙酸水解酶或α-1抗胰蛋白酶的功能。CRISPR还被研究用于治疗乙型肝炎感染。CRISPR是一个令人兴奋的、不断发展的技术，它对肝脏疾病的基因特征和发现起到了推动作用，并具有安全和永久治疗肝脏疾病的潜力。版权所有©2023年美国肝病研究协会。

CRISPR is a gene editing tool adapted from naturally occurring defense systems from bacteria. It is a technology that is revolutionizing the interrogation of gene functions in driving liver disease, especially through genetic screens and by facilitating animal knockout and knockin models. It is being used in models of liver disease to identify which genes are critical for liver pathology, especially in genetic liver disease, hepatitis, and in cancer initiation and progression. It holds tremendous promise in treating human diseases directly by editing DNA. It could disable gene function in the case of expression of a maladaptive protein, such as blocking transthyretin as a therapy for amyloidosis, or to correct gene defects, such as restoring the normal functions of liver enzymes fumarylacetoacetate hydrolase or alpha-1 antitrypsin. It is also being studied for treatment of hepatitis B infection. CRISPR is an exciting, evolving technology that is facilitating gene characterization and discovery in liver disease and holds the potential to treat liver diseases safely and permanently.Copyright © 2023 American Association for the Study of Liver Diseases.