Induced Pluripotent Stem Cells and Their Applications in Amyotrophic Lateral Sclerosis

Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease that results in the loss of motor function in the central nervous system (CNS) and ultimately death. The mechanisms underlying ALS pathogenesis have not yet been fully elucidated, and ALS cannot be treated effectively. Mo...

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Published inCells (Basel, Switzerland) Vol. 12; no. 6; p. 971
Main Authors Du, Hongmei, Huo, Zijun, Chen, Yanchun, Zhao, Zhenhan, Meng, Fandi, Wang, Xuemei, Liu, Shiyue, Zhang, Haoyun, Zhou, Fenghua, Liu, Jinmeng, Zhang, Lingyun, Zhou, Shuanhu, Guan, Yingjun, Wang, Xin
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Abstract Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease that results in the loss of motor function in the central nervous system (CNS) and ultimately death. The mechanisms underlying ALS pathogenesis have not yet been fully elucidated, and ALS cannot be treated effectively. Most studies have applied animal or single-gene intervention cell lines as ALS disease models, but they cannot accurately reflect the pathological characteristics of ALS. Induced pluripotent stem cells (iPSCs) can be reprogrammed from somatic cells, possessing the ability to self-renew and differentiate into a variety of cells. iPSCs can be obtained from ALS patients with different genotypes and phenotypes, and the genetic background of the donor cells remains unchanged during reprogramming. iPSCs can differentiate into neurons and glial cells related to ALS. Therefore, iPSCs provide an excellent method to evaluate the impact of diseases on ALS patients. Moreover, patient-derived iPSCs are obtained from their own somatic cells, avoiding ethical concerns and posing only a low risk of immune rejection. The iPSC technology creates new hope for ALS treatment. Here, we review recent studies on iPSCs and their applications in disease modeling, drug screening and cell therapy in ALS, with a particular focus on the potential for ALS treatment.
AbstractList Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease that results in the loss of motor function in the central nervous system (CNS) and ultimately death. The mechanisms underlying ALS pathogenesis have not yet been fully elucidated, and ALS cannot be treated effectively. Most studies have applied animal or single-gene intervention cell lines as ALS disease models, but they cannot accurately reflect the pathological characteristics of ALS. Induced pluripotent stem cells (iPSCs) can be reprogrammed from somatic cells, possessing the ability to self-renew and differentiate into a variety of cells. iPSCs can be obtained from ALS patients with different genotypes and phenotypes, and the genetic background of the donor cells remains unchanged during reprogramming. iPSCs can differentiate into neurons and glial cells related to ALS. Therefore, iPSCs provide an excellent method to evaluate the impact of diseases on ALS patients. Moreover, patient-derived iPSCs are obtained from their own somatic cells, avoiding ethical concerns and posing only a low risk of immune rejection. The iPSC technology creates new hope for ALS treatment. Here, we review recent studies on iPSCs and their applications in disease modeling, drug screening and cell therapy in ALS, with a particular focus on the potential for ALS treatment.
Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease that results in the loss of motor function in the central nervous system (CNS) and ultimately death. The mechanisms underlying ALS pathogenesis have not yet been fully elucidated, and ALS cannot be treated effectively. Most studies have applied animal or single-gene intervention cell lines as ALS disease models, but they cannot accurately reflect the pathological characteristics of ALS. Induced pluripotent stem cells (iPSCs) can be reprogrammed from somatic cells, possessing the ability to self-renew and differentiate into a variety of cells. iPSCs can be obtained from ALS patients with different genotypes and phenotypes, and the genetic background of the donor cells remains unchanged during reprogramming. iPSCs can differentiate into neurons and glial cells related to ALS. Therefore, iPSCs provide an excellent method to evaluate the impact of diseases on ALS patients. Moreover, patient-derived iPSCs are obtained from their own somatic cells, avoiding ethical concerns and posing only a low risk of immune rejection. The iPSC technology creates new hope for ALS treatment. Here, we review recent studies on iPSCs and their applications in disease modeling, drug screening and cell therapy in ALS, with a particular focus on the potential for ALS treatment.Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease that results in the loss of motor function in the central nervous system (CNS) and ultimately death. The mechanisms underlying ALS pathogenesis have not yet been fully elucidated, and ALS cannot be treated effectively. Most studies have applied animal or single-gene intervention cell lines as ALS disease models, but they cannot accurately reflect the pathological characteristics of ALS. Induced pluripotent stem cells (iPSCs) can be reprogrammed from somatic cells, possessing the ability to self-renew and differentiate into a variety of cells. iPSCs can be obtained from ALS patients with different genotypes and phenotypes, and the genetic background of the donor cells remains unchanged during reprogramming. iPSCs can differentiate into neurons and glial cells related to ALS. Therefore, iPSCs provide an excellent method to evaluate the impact of diseases on ALS patients. Moreover, patient-derived iPSCs are obtained from their own somatic cells, avoiding ethical concerns and posing only a low risk of immune rejection. The iPSC technology creates new hope for ALS treatment. Here, we review recent studies on iPSCs and their applications in disease modeling, drug screening and cell therapy in ALS, with a particular focus on the potential for ALS treatment.
Audience Academic
Author Zhao, Zhenhan
Zhang, Haoyun
Wang, Xuemei
Wang, Xin
Zhou, Shuanhu
Meng, Fandi
Liu, Jinmeng
Du, Hongmei
Liu, Shiyue
Chen, Yanchun
Guan, Yingjun
Huo, Zijun
Zhou, Fenghua
Zhang, Lingyun
AuthorAffiliation 5 Department of Neurosurgery, Brigham and Women’s Hospital, Harvard Medical School, Boston, MA 02115, USA
3 Department of Pathology, School of Basic Medical Sciences, Weifang Medical University, Weifang 261053, China
4 Harvard Medical School and Harvard Stem Cell Institute, Harvard University, Boston, MA 02115, USA; shuanhuzhou@gmail.com
1 Department of Histology and Embryology, School of Basic Medical Sciences, Weifang Medical University, Weifang 261053, China; hongmeiduwf@163.com (H.D.); zijunhuo1998@163.com (Z.H.); cyc7907@wfmc.edu.cn (Y.C.); jinqiuhupan@163.com (Z.Z.); 18800460605@163.com (F.M.); 18863662168@163.com (X.W.)
2 Neurologic Disorders and Regenerative Repair Laboratory, Weifang Medical University, Weifang 261053, China; liu08308399@163.com (S.L.); haoyunzh@163.com (H.Z.); zhoufh@wfmc.edu.cn (F.Z.); 18353687185@163.com (J.L.); zly199311@126.com (L.Z.)
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Keywords induced pluripotent stem cells
drug screening
amyotrophic lateral sclerosis
cell therapy
disease modeling
Language English
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These authors contributed equally to this work.
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Snippet Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease that results in the loss of motor function in the central nervous system (CNS)...
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SubjectTerms Amyotrophic lateral sclerosis
Amyotrophic Lateral Sclerosis - genetics
Amyotrophic Lateral Sclerosis - metabolism
Amyotrophic Lateral Sclerosis - therapy
Animals
Care and treatment
Cell differentiation
Cell lines
Cell therapy
Cell- and Tissue-Based Therapy
Cellular therapy
Central nervous system
Development and progression
Disease
disease modeling
Drug screening
Efficiency
Family medical history
Fibroblasts
Genotypes
Glial cells
Health aspects
induced pluripotent stem cells
Induced Pluripotent Stem Cells - metabolism
Inhibitory postsynaptic potentials
Methods
Mutation
Nervous system
Neurodegenerative diseases
Neurodegenerative Diseases - metabolism
Neuronal-glial interactions
Neurons - metabolism
Oxidative stress
Pathogenesis
Phenotypes
Physiological aspects
Pluripotency
Proteins
Review
Somatic cells
Stem cells
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Title Induced Pluripotent Stem Cells and Their Applications in Amyotrophic Lateral Sclerosis
URI https://www.ncbi.nlm.nih.gov/pubmed/36980310
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Volume 12
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