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Abstract
Neurodegenerative diseases are characterized by a gradual loss of cognitive and physical functions. Medications for these disorders are limited and treat the symptoms only. There are no disease-modifying therapies available, which have been shown to slow or stop the continuing loss of neurons. Transdifferentiation, whereby somatic cells are reprogrammed into another lineage without going through an intermediate proliferative pluripotent stem cell stage, provides an alternative strategy for regenerative medicine and disease modeling. In particular, the transdifferentiation of somatic cells into specific subset of patient-specific neuronal cells offers alternative autologous cell therapeutic strategies for neurodegenerative disorders and presents a rich source of using diverse somatic cell types for relevant applications in translational, personalized medicine, as well as human mechanistic study, new drug-target identification, and novel drug screening systems. Here, we provide a comprehensive overview of the recent development of transdifferentiation research, with particular attention to chemical-induced transdifferentiation and perspectives for modeling and treatment of neurodegenerative diseases.
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Details
1 Department of Neuroscience, Istituto Superiore di Sanità, Rome, Italy; Institute of Translational Pharmacology, National Research Council, Rome, Italy
2 Translational Medical Center for Stem Cell Therapy, Shanghai East Hospital, School of Medicine, Shanghai, China; School of Life Science and Technology, Tongji University, Shanghai, China
3 IRCCS San Raffaele Pisana, Rome, Italy
4 IRCCS San Raffaele Pisana, Rome, Italy; University San Raffaele, Rome, Italy
5 Department of Neuroscience, Istituto Superiore di Sanità, Rome, Italy
6 School of Life Science and Technology, Tongji University, Shanghai, China; State Key Laboratory of Cell Biology, CAS Center for Excellence in Molecular Cell Science, Shanghai Institute of Biochemistry and Cell Biology, Chinese Academy of Sciences, Shanghai, P. R. China