摘要
Rett syndrome(RTT)is a progressive neurodevelopmental disorder that occurs mainly in girls with a range of typical symptoms of autism spectrum disorders.MeCP2 protein loss-of-function in neural lineage cells is the main cause of RTT pathogenicity.As it is still hard to understand the mechanism of RTT on the basis of only clinical patients or animal models,cell models cultured in vitro play indispensable roles.Here we reviewed the research progress in the pathogenesis of RTT at the cellular level,summarized the preclinical-research-related applications,and prospected potential future development.
基金
The Major Basic Research Project of Science and Technology of Yunnan
Grant/Award Number:202001BC070001 and 202105AC160041
National Natural Science Foundation of China
Grant/Award Number:81930121 and 31960120
The National Key Research and Development Program of China
Grant/Award Number:2018YFA0107902 and 2018YFA0801403。