| 遗传病的生物治疗 |
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| 引用本文： | 杨京,谢杨丽,陈林.遗传病的生物治疗[J].生物工程学报,2015,31(6):968-975. |
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| 作者姓名： | 杨京 谢杨丽 陈林 |
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| 作者单位： | 第三军医大学大坪医院 创伤、烧伤与复合伤国家重点实验室骨代谢与修复中心 创伤实验室 康复理疗科，重庆 400042,第三军医大学大坪医院 创伤、烧伤与复合伤国家重点实验室骨代谢与修复中心 创伤实验室 康复理疗科，重庆 400042,第三军医大学大坪医院 创伤、烧伤与复合伤国家重点实验室骨代谢与修复中心 创伤实验室 康复理疗科，重庆 400042 |
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| 基金项目： | 国家自然科学基金 (No. 81220108020)，重庆市基础与前沿研究计划项目 (No. CSTC2013jcyjC00009) 资助。 |
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| 摘 要： | 遗传病尤其是单基因遗传病是儿童致畸致残的主要原因之一,给家庭、社会带来沉重的经济、心理负担。目前绝大多数遗传病临床治疗以对症治疗为主,尚缺乏有效的治疗方法。随着生命与医学科学的发展,近年来以靶向致病分子或其相关信号通路、靶分子的外源物质补充、转基因、RNA干扰、基因组编辑等生物学技术为代表的生物学治疗措施开始应用于遗传病的治疗,并取得了一定的疗效。但目前大多数遗传病的生物学治疗仍局限于动物实验研究,今后在继续研发基于致病机制的新治疗策略的同时,将关注如何开展临床试验,为最终安全、有效应用于临床患者奠定基础。
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| 关 键 词： | 单基因遗传病，RNA干扰，基因组编辑 |
| 收稿时间： | 2015/1/15 0:00:00 |
Biologic treatments for hereditary diseases |
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| Jing Yang,Yangli Xie and Lin Chen.Biologic treatments for hereditary diseases[J].Chinese Journal of Biotechnology,2015,31(6):968-975. |
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| Authors: | Jing Yang Yangli Xie and Lin Chen |
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| Institution: | Department of Rehabilitation and Physiotherapy, Trauma laboratory, Center of Bone Metabolism and Repair, State Key Laboratory of Trauma Burns and Combined Injury, Institute of Surgery Research Daping Hospital, Third Military Medical University, Chongqing 400042, China,Department of Rehabilitation and Physiotherapy, Trauma laboratory, Center of Bone Metabolism and Repair, State Key Laboratory of Trauma Burns and Combined Injury, Institute of Surgery Research Daping Hospital, Third Military Medical University, Chongqing 400042, China and Department of Rehabilitation and Physiotherapy, Trauma laboratory, Center of Bone Metabolism and Repair, State Key Laboratory of Trauma Burns and Combined Injury, Institute of Surgery Research Daping Hospital, Third Military Medical University, Chongqing 400042, China |
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| Abstract: | Hereditary disease, especially monogenic disease is one of the major causes for malformation and disability of children. Most hereditary diseases have no effective therapy besides clinical symptomatic treatment. Biological techniques targeting casual genes or related signaling genes, such as transgenic, RNA interfere, genome editing, have been successfully applied in treating some hereditary diseases. However, most biological treatments were carried out in animals, further confirmation of the effectiveness and safety of these therapies, and development of more therapeutic approaches based on mechanisms are needed before clinical trials. |
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| Keywords: | monogenic disease RNAi genome editing |
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