Sci Adv: The Tulip Tai team at Huashan Hospital found new targets for the prevention and treatment of neurodegenerative diseases

PGRN (progranulin) is a secretion-type multi-functional glycoprotein, distributed in the central nervous system and peripheral, involved in neurodevelopment, regeneration, nerve inflammation, autophagy and other life activities, PGRN dysfunction can lead to a variety of neurodegenerative diseases.
Recently, Professor Tulip Tai, Professor Dong Qiang of Huashan Hospital, affiliated with Fudan University, in collaboration with Professor Tan Lan of Qingdao Municipal Hospital affiliated with Qingdao University, Professor Zhang Can of The Massachusetts General Hospital of Harvard University, and Professor Wang Yanjiang of Daping Hospital of the Army Medical University, published an online research paper entitled: The FAM171A2 gene gene a key of the pronulin expression and the study of the relationship.
Based on clinical research, bioinficial analysis, and basic experiments, this study found for the first time that the new gene FAM171A2 is a key regulatory gene for the important protein particle protein prelude (progranulin, PGRN) associated with the onset of neurodegenerative diseases, and is a risk gene for neurodegenerative diseases such as Alzheimer's disease (AD), Parkinson's disease (PD), and frontal lobe dementia (FTLD).
findings could help shed light on the regulatory networks of PGRN in the brain and provide new targets for the prevention and treatment of neurodegenerative diseases.
this study confirmed for the first time in clinical studies that the functional polymorphism of the FAM171A2 gene was significantly associated with the level of PGRN in cerebrospinal fluid, explaining its 9.1% variability, and that the expression level of FAM171A2 in in-brain and exomensective blood was also significantly correlated with the level of PGRN expression.
further demonstrated that raising the expression level of FAM171A2 inhibits PGRN production and secretion through double luciferase reporting experiments, gene silencing/over-expression in-body experiments.
THE FAM171A2 gene has also been found to be a risk gene for neurodegenerative diseases such as Alzheimer's disease (AD), Parkinson's disease (PD), frontal temporal lobe dementia (FTLD).
addition, the study made clear that FAM171A2 is expressed on vascular endothrotytes and small glial cells, suggesting that the gene may have the function of regulating blood-brain barriers and nerve inflammation.
, FAM171A2, by regulating PGRN, affects the risk of neurodegenerative diseases and is a potential new target for the prevention and treatment of neurodegenerative diseases.