Modeling Human Neurological Diseases

      The limited access to patient neurons greatly impedes the progress of research in neurological diseases. Reprogramming of human neurons from adult fibroblasts provides an unprecedented approach to deciphering the molecular pathogenesis underlying disease conditions. Using lentiviral delivery of transcription factors, we can generate human neurons from adult fibroblast via two strategies. One strategy is the direct conversion of fibroblasts into neurons (for example, directly induced motor neurons, diMNs). The other strategy is induced pluripotent stem cells (iPSCs)-based reprogramming and differentiation (for example, iPSC-MNs). Using these patient-specific neurons, research efforts are focusing on the molecular and cellular pathophysiology of neurological disorders, such as Alzheimer disease (AD), Dystonia and amyotrophic lateral sclerosis (ALS).

Selected Publications

Baojin Ding*, Yu Tang, Shuaipeng Ma, Meng-Lu Liu, Tong Zang, Masuma Akter and Chun-Li Zhang (2020). Disease modeling with human neurons reveals LMNB1 dysregulation underlying DYT1 dystonia. bioRxiv. doi:

Sepehrimanesh M, and Ding B* (2020). Generation and Optimization of Highly Pure Motor Neurons from Human Induced Pluripotent Stem Cells via Lentiviral Delivery of Transcription Factors. Am J Physiol Cell Physiol. 2020 Aug 12. doi: 10.1152/ajpcell.00279.2020. PMID: 32783653

Ding B, Akter M and Zhang C-L. (2020). Differential Influence of Sample Sex and Neuronal Maturation on mRNA and Protein Transport in Induced Human Neurons. Front. Mol. Neurosci. 13:46 doi: 10.3389/fnmol. 2020.00046


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