Diagnosis and drug screening of molecular signature of early onset sporadic Parkinson's disease

摘要

Artificial pluripotent stem cell (IPSC) technology allows the formation and research of living brain tissue associated with Parkinson's disease (PD) in vivo.By using cell line derived from PD patient, a powerful discovery system which combines the phenotype of the cell observed in vitro with actual clinical data is realized.By differentiating IPSC from patients to dopaminergic (DA) nerve fate, we found that these cells exhibit molecular and functional properties of DA neurons, which are observed to be significantly altered in the substantia nigra of PD patients.A clinical manifestation driving the generation of other related cell types can also result in a novel PD specific phenotype in vitro that may lead to a new therapeutic approach for patients with PD.The differentiated neural tissue from these patients is due to the early onset and non familial origin of the patients to discover neuronal subtype specific pathogenesis mechanisms, and We provide an important opportunity to investigate the contribution of patients' genetic backgrounds to susceptibility to PD.