Application of live biotherapeutic products and perspective in the treatment of inherited metabolic disease.
- Author:
Zhaowei CHEN
1
;
Min GUO
1
;
Huajun ZHENG
1
Author Information
- Publication Type:Journal Article
- Keywords: engineered strain; gene editing; inherited metabolic disease; live biotherapeutic products (LBPs); synthetic biology
- MeSH: Humans; Bacteria/genetics*; Gene Editing; Metabolic Diseases/therapy*
- From: Chinese Journal of Biotechnology 2023;39(4):1290-1303
- CountryChina
- Language:Chinese
- Abstract: 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.
