Pluripotent Stem Cells for Modelling and Cell Therapy of Parkinson’s Disease

• Published in: Biochemistry (Moscow) Vol. 83; no. 9; pp. 1046 - 1056
• Main Authors: Lebedeva, O. S., Lagarkova, M. A.
• Format: Journal Article
• Language: English
• Published: Moscow Pleiades Publishing 01-09-2018; Springer; Springer Nature B.V
• Subjects: Animal models; Animals; Biochemistry; Biomedical and Life Sciences; Biomedicine; Bioorganic Chemistry; Care and treatment; Cell Differentiation; Cell- and Tissue-Based Therapy; Development and progression; Disease; Dopamine receptors; Dopaminergic Neurons - cytology; Dopaminergic Neurons - transplantation; Drug screening; Health aspects; Human Embryonic Stem Cells - cytology; Human Embryonic Stem Cells - metabolism; Humans; Induced Pluripotent Stem Cells - cytology; Induced Pluripotent Stem Cells - metabolism; Inhibitory postsynaptic potentials; Laboratory animals; Laboratory tests; Leucine-Rich Repeat Serine-Threonine Protein Kinase-2 - genetics; Life Sciences; Microbiology; Molecular modelling; Movement disorders; Neurodegeneration; Neurodegenerative diseases; Neuroprotection; Parkinson disease; Parkinson Disease - pathology; Parkinson Disease - therapy; Parkinson's disease; Pluripotency; Review; Stem cell transplantation; Stem cells; Therapeutic applications; Ubiquitin-Protein Ligases - genetics; induced pluripotent stem cells; cell therapy; isogenic system; Parkinson’s disease; cell model; dopaminergic neurons
• ISSN: 0006-2979; 1608-3040
• DOI: 10.1134/S0006297918090067

Studying pathogenesis of neurodegenerative diseases, including Parkinson’s disease (PD), requires adequate disease models. The available patient’s material is limited to biological fluids and
post mortem
brain samples. Disease modeling and drug screening can be done in animal models, although this approach has its own limitations, since laboratory animals do not suffer from many neurodegenerative diseases, including PD. The use of neurons obtained by targeted differentiation from induced pluripotent stem cells (iPSCs) with known genetic mutations, as well as from carriers of sporadic forms of the disease, will allow to elucidate new components of the molecular mechanisms of neurodegeneration. Such neuronal cultures can also serve as unique models for testing neuroprotective compounds and monitoring neurodegenerative changes against a background of various therapeutic interventions. In the future, dopaminergic neurons differentiated from iPSCs can be used for cell therapy of PD.