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Targeting the Liver with Nucleic Acid Therapeutics for the Treatment of Systemic Diseases of Liver Origin
Pharmacological Reviews ( IF 19.3 ) Pub Date : 2024-01-01 , DOI: 10.1124/pharmrev.123.000815 Anagha Gogate 1 , Jordyn Belcourt 1 , Milan Shah 1 , Alicia Zongxun Wang 1 , Alexis Frankel 1 , Holly Kolmel 1 , Matthew Chalon 1 , Prajith Stephen 1 , Aarush Kolli 1 , Sherouk M Tawfik 1 , Jing Jin 1 , Raman Bahal 1 , Theodore P Rasmussen 1 , José E Manautou 1 , Xiao-Bo Zhong 2
Pharmacological Reviews ( IF 19.3 ) Pub Date : 2024-01-01 , DOI: 10.1124/pharmrev.123.000815 Anagha Gogate 1 , Jordyn Belcourt 1 , Milan Shah 1 , Alicia Zongxun Wang 1 , Alexis Frankel 1 , Holly Kolmel 1 , Matthew Chalon 1 , Prajith Stephen 1 , Aarush Kolli 1 , Sherouk M Tawfik 1 , Jing Jin 1 , Raman Bahal 1 , Theodore P Rasmussen 1 , José E Manautou 1 , Xiao-Bo Zhong 2
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Systemic diseases of liver origin (SDLO) are complex diseases in multiple organ systems, such as cardiovascular, musculoskeletal, endocrine, renal, respiratory, and sensory organ systems, caused by irregular liver metabolism and production of functional factors. Examples of such diseases discussed in this article include primary hyperoxaluria, familial hypercholesterolemia, acute hepatic porphyria, hereditary transthyretin amyloidosis, hemophilia, atherosclerotic cardiovascular diseases, α-1 antitrypsin deficiency-associated liver disease, and complement-mediated diseases. Nucleic acid therapeutics use nucleic acids and related compounds as therapeutic agents to alter gene expression for therapeutic purposes. The two most promising, fastest-growing classes of nucleic acid therapeutics are antisense oligonucleotides (ASOs) and small interfering RNAs (siRNAs). For each listed SDLO disease, this article discusses epidemiology, symptoms, genetic causes, current treatment options, and advantages and disadvantages of nucleic acid therapeutics by either ASO or siRNA drugs approved or under development. Furthermore, challenges and future perspectives on adverse drug reactions and toxicity of ASO and siRNA drugs for the treatment of SDLO diseases are also discussed. In summary, this review article will highlight the clinical advantages of nucleic acid therapeutics in targeting the liver for the treatment of SDLO diseases.
中文翻译：
用核酸治疗靶向肝脏治疗肝源性系统性疾病
肝源性系统性疾病（SDLO）是由肝脏代谢和功能因子产生异常引起的心血管、肌肉骨骼、内分泌、肾脏、呼吸和感觉器官系统等多器官系统的复杂疾病。本文讨论的此类疾病的例子包括原发性高草酸尿症、家族性高胆固醇血症、急性肝卟啉症、遗传性运甲状腺素蛋白淀粉样变性、血友病、动脉粥样硬化性心血管疾病、 α -1 抗胰蛋白酶缺乏相关的肝病和补体介导的疾病。核酸疗法使用核酸和相关化合物作为治疗剂来改变基因表达以达到治疗目的。最有前途、增长最快的两类核酸治疗药物是反义寡核苷酸 (ASO) 和小干扰 RNA (siRNA)。对于每种列出的 SDLO 疾病，本文讨论了流行病学、症状、遗传原因、当前的治疗方案以及已批准或正在开发的 ASO 或 siRNA 药物的核酸疗法的优缺点。此外，还讨论了 ASO 和 siRNA 药物治疗 SDLO 疾病的药物不良反应和毒性的挑战和未来前景。总之，这篇综述文章将重点介绍核酸疗法在靶向肝脏治疗 SDLO 疾病方面的临床优势。
更新日期：2023-12-16
中文翻译：
用核酸治疗靶向肝脏治疗肝源性系统性疾病
肝源性系统性疾病（SDLO）是由肝脏代谢和功能因子产生异常引起的心血管、肌肉骨骼、内分泌、肾脏、呼吸和感觉器官系统等多器官系统的复杂疾病。本文讨论的此类疾病的例子包括原发性高草酸尿症、家族性高胆固醇血症、急性肝卟啉症、遗传性运甲状腺素蛋白淀粉样变性、血友病、动脉粥样硬化性心血管疾病、 α -1 抗胰蛋白酶缺乏相关的肝病和补体介导的疾病。核酸疗法使用核酸和相关化合物作为治疗剂来改变基因表达以达到治疗目的。最有前途、增长最快的两类核酸治疗药物是反义寡核苷酸 (ASO) 和小干扰 RNA (siRNA)。对于每种列出的 SDLO 疾病，本文讨论了流行病学、症状、遗传原因、当前的治疗方案以及已批准或正在开发的 ASO 或 siRNA 药物的核酸疗法的优缺点。此外，还讨论了 ASO 和 siRNA 药物治疗 SDLO 疾病的药物不良反应和毒性的挑战和未来前景。总之，这篇综述文章将重点介绍核酸疗法在靶向肝脏治疗 SDLO 疾病方面的临床优势。
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