Chen Zhaowei, Guo Min, Zheng Huajun
National Health Commission Key Laboratory of Reproduction Regulation, Shanghai Institute for Biomedical and Pharmaceutical Technologies, Fudan University, Shanghai 200032, China.
State Key Laboratory of Bioreactor Engineering, New World Institute of Biotechnology, East China University of Science and Technology, Shanghai 200237, China.
Sheng Wu Gong Cheng Xue Bao. 2023 Apr 25;39(4):1290-1303. doi: 10.13345/j.cjb.220603.
Live biotherapeutic products (LBPs) refer to the living bacteria derived from human body intestinal gut or in nature that can be used to treat the human disease. However, the naturally screened living bacteria have some disadvantages, such as deficient therapeutic effect and great divergence, which fall short of the personalized diagnosis and treatment needs. In recent years, with the development of synthetic biology, researchers have designed and constructed several engineered strains that can respond to external complex environmental signals, which speeded up the process of development and application of LBPs. Recombinant LBPs modified by gene editing can have therapeutic effect on specific diseases. Inherited metabolic disease is a type of disease that causes a series of clinical symptoms due to the genetic defect of some enzymes in the body, which may cause abnormal metabolism the corresponding metabolites. Therefore, the use of synthetic biology to design LBPs targeting specific defective enzymes will be promising for the treatment of inherited metabolic defects in the future. This review summarizes the clinic applications of LBPs and its potential for the treatment of inherited metabolic defects.
活体生物治疗产品（LBPs）是指来源于人体肠道或自然界的可用于治疗人类疾病的活细菌。然而，天然筛选出的活细菌存在一些缺点，如治疗效果不足和差异较大，无法满足个性化诊断和治疗需求。近年来，随着合成生物学的发展，研究人员设计并构建了几种能够响应外部复杂环境信号的工程菌株，这加速了LBPs的开发和应用进程。通过基因编辑修饰的重组LBPs可对特定疾病产生治疗作用。遗传性代谢疾病是一类由于体内某些酶的基因缺陷导致一系列临床症状的疾病，这可能会导致相应代谢产物的代谢异常。因此，利用合成生物学设计针对特定缺陷酶的LBPs在未来治疗遗传性代谢缺陷方面具有广阔前景。本综述总结了LBPs的临床应用及其治疗遗传性代谢缺陷的潜力。
