Abstract
Many human neurological diseases are not currently curable and result in devastating neurologic sequelae. The increasing availability of induced pluripotent stem cells (iPSCs) derived from adult human somatic cells provides new prospects for cellreplacement strategies and disease-related basic research in a broad spectrum of human neurologic diseases. Patient-specific iPSC-based modeling of neurogenetic and neurodegenerative diseases is an emerging efficient tool for in vitro modeling to understand disease and to screen for genes and drugs that modify the disease process. With the exponential increase in iPSC research in recent years, human iPSCs have been successfully derived with different technologies and from various cell types. Although there remain a great deal to learn about patient-specific iPSC safety, the reprogramming mechanisms, better ways to direct a specific reprogramming, ideal cell source for cellular grafts, and the mechanisms by which transplanted stem cells lead to an enhanced functional recovery and structural reorganization, the discovery of the therapeutic potential of iPSCs offers new opportunities for the treatment of incurable neurologic diseases. However, iPSC-based therapeutic strategies need to be thoroughly evaluated in preclinical animal models of neurological diseases before they can be applied in a clinical setting.
Highlights
Human neurological diseases including stroke, neurodegenerative disorders, neurotrauma, multiple sclerosis (MS), and neurodevelopmental disorders are caused by a loss of neurons and glial cells in the brain or spinal cord
Critical steps in induced pluripotent stem cells (iPSCs) generation include that reprogramming factors can be efficiently overexpressed in the somatic cell type that is chosen for reprogramming and that expression of the exogenously applied reprogramming factors is silenced once the cells have been transformed to a pluripotent stage
Incomplete disease modeling using iPSCs has first been reported in autosomal recessive neurodegenerative diseases by Ebert et al it showed that spinal muscular atrophy (SMA)-iPSCs-derived neural cells undergo selective neuronal death, which could be reversed by adding the compounds known to raise the production of survival motor neuron (SMN) protein [91]
Summary
Human neurological diseases including stroke, neurodegenerative disorders, neurotrauma, multiple sclerosis (MS), and neurodevelopmental disorders are caused by a loss of neurons and glial cells in the brain or spinal cord. They usually cause morbidity and mortality as well as increase social and economic burdens of patients and their caregivers [1]. Neural stem cells (NSCs) are multipotent stem cells which are derived from neural tissues [9] These cells are self-renewing and differentiate into lineage-specific neural precursor or progenitor cells (NPCs) that can give rise to all cell types (neurons, astrocytes, and oligodendrocyes) of the nervous system through asymmetric cell division. We summarized the recent advancements in iPSC generation, their capacity for differentiation toward neural lineages, and iPSC-based transplantation and disease modeling attempts for neurological diseases
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