A Comprehensive Library of Familial Human Amyotrophic Lateral Sclerosis Induced Pluripotent Stem Cells

Liu, Ying; Balasubramanian, Umamahesw; Cohen, Devon; Zhang, Ping Wu; Mosmiller, Elizabeth; Sattler, Rita; Maragakis, Nicholas J.; Rothstein, Jeffrey D.

doi:10.1371/journal.pone.0118266

Cited by 39 publications

(37 citation statements)

References 63 publications

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“…ALS is a devastating disease; the loss of motor neurons and muscle atrophy confine patients to a wheelchair very rapidly, followed by respiratory failure. The cause of ALS is not yet elucidated, however, mutations in genes SOD1, C9orf72, TDP-43, FUS/TLS, angiogenin, Matrin 3 [60][61][62][63][64][65] and others, have been associated with ALS. Moreover, familial inheritance accounts for about 10% of the cases of patients diagnosed with ALS [65] .…”

Section: Alsmentioning

confidence: 99%

“…The cause of ALS is not yet elucidated, however, mutations in genes SOD1, C9orf72, TDP-43, FUS/TLS, angiogenin, Matrin 3 [60][61][62][63][64][65] and others, have been associated with ALS. Moreover, familial inheritance accounts for about 10% of the cases of patients diagnosed with ALS [65] . Several studies using reprogrammed cells generated from patients of different diseases have been described since 2008 [66,67] and they have and still contribute to the understanding, from a physiological point of view to prospective treatments, of these diseases.…”

Section: Alsmentioning

confidence: 99%

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Induced pluripotent stem cells for modeling neurological disorders

Russo

Cugola

Fernandes

et al. 2015

WJT

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Section: Alsmentioning

confidence: 99%

Section: Alsmentioning

confidence: 99%

Induced pluripotent stem cells for modeling neurological disorders

Russo

Cugola

Fernandes

et al. 2015

WJT

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“…Neural stem cells or MNs derived from ALS patients or bearing ALS mutations could be used in long-term cell cultures or co-culture with glial cells to examine pathophysiological hallmarks of the disease, including analysis of protein aggregates, altered pathways in the neurodegenerative process, in particular the imbalance of mitochondrial dynamics, electrophysiological properties, gene expression analyses, and examination of patterns of alternative pre-mRNA splicing. These studies might provide important insights into ALS, and provide cell-based assays for drug screening (Bilican et al, 2012; de Boer et al, 2014; Di Giorgio et al, 2008; Dimos et al, 2008; Donnelly et al, 2013; Kiskinis et al, 2014; Li et al, 2015b; Marchetto et al, 2008; Sareen et al, 2013; Wainger et al, 2014). …”

Section: Disease Modeling For Als Using Human Pluripotent Stem Celmentioning

confidence: 99%

Reverse engineering human neurodegenerative disease using pluripotent stem cell technology

Liu

Deng

2016

Brain Research

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With the technology of reprogramming somatic cells by introducing defined transcription factors that enables the generation of “induced pluripotent stem cells (iPSCs)” with pluripotency comparable to that of embryonic stem cells (ESCs), it has become possible to use this technology to produce various cells and tissues that have been difficult to obtain from living bodies. This advancement is bringing forth rapid progress in iPSC-based disease modeling, drug screening, and regenerative medicine. More and more studies have demonstrated that phenotypes of adult-onset neurodegenerative disorders could be rather faithfully recapitulated in iPSC-derived neural cell cultures. Moreover, despite the adult-onset nature of the diseases, pathogenic phenotypes and cellular abnormalities often exist in early developmental stages, providing new “windows of opportunity” for understanding mechanisms underlying neurodegenerative disorders and for discovering new medicines. The cell reprogramming technology enables a reverse engineering approach for modeling the cellular degenerative phenotypes of a wide range of human disorders. An excellent example is the study of the human neurodegenerative disease amyotrophic lateral sclerosis (ALS) using iPSCs. ALS is a progressive neurodegenerative disease characterized by the loss of upper and lower motor neurons (MNs), culminating in muscle wasting and death from respiratory failure. The iPSC approach provides innovative cell culture platforms to serve as ALS patient-derived model systems. Researchers have converted iPSCs derived from ALS patients into MNs and various types of glial cells, all of which are involved in ALS, to study the disease. The iPSC technology could be used to determine the role of specific genetic factors to track down what’s wrong in the neurodegenerative disease process in the “disease-in-a-dish” model. Meanwhile, parallel experiments of targeting the same specific genes in human ESCs could also be performed to control and to complement the iPSC-based approach for ALS disease modeling studies. Much knowledge has been generated from the study of both ALS iPSCs and ESCs. As these methods have advantages and disadvantages that should be balanced on experimental design in order for them to complement one another, combining the diverse methods would help build an expanded knowledge of ALS pathophysiology. The goals are to reverse engineer the human disease using ESCs and iPSCs, generate lineage reporter lines and in vitro disease models, target disease related genes, in order to better understand the molecular and cellular mechanisms of differentiation regulation along neural (neuronal versus glial) lineages, to unravel the pathogenesis of the neurodegenerative disease, and to provide appropriate cell sources for replacement therapy.

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“…In-vitro studies have proven the directed conversion of mesenchymal stem cells into functional motor neuron-like cells as a step towards transplantation in motor neuron disease (38) Alzheimer disease (AD) Alzheimer disease (AD) is due to deposition of amyloid fibrils that consists of Amyloid beta (Abeta) proteins, therefore inhibition of Abeta formation by transthyretin, a natural neuroprotector protein in CSF, or Abeta-transthyretin complexes mimetic should inhibit amyloid formation as proved invitro. (39) A neural compound come from algae called homotaurine (tramiprosate)has been tested invitro and now in phase 3 clinical study to be beneficial preventing AD owing to its anti-amyloid activity and gamma-amino butyric acid type A receptor affinity.…”

Section: In-vitro Trials Involving Mechanisms Of Stem Cell-mentioning

confidence: 99%

Stem Cells for Neuro-regeneration: State of the Art

Wagih¹,

Elhawary²,

Ellessy³

et al. 2015

BIO

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Neuroregeneration (NR) is a long-sought medical dream which has intrigued vast research in the past decades. A traditional physiologic dogma that central nervous system does not regenerate has been strongly challenged in the recent years since the advent of the stem cell era. Stem cell research in the regenerative field passes through three main stages. The first stage is the in-vitro experiments which studies the exact molecular and cellular mechanisms underlying stem cell-mediated NR. The second stage is the application of these data in experimental animal settings to provide "proof of concept" of stem cell therapy in animal models. The final step is the translation of these data in pilot clinical trials. In this review, we will try to gather the different data of stem cell-mediated NR from various experimental and clinical researches.

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A Comprehensive Library of Familial Human Amyotrophic Lateral Sclerosis Induced Pluripotent Stem Cells

Cited by 39 publications

References 63 publications

Induced pluripotent stem cells for modeling neurological disorders

Induced pluripotent stem cells for modeling neurological disorders

Reverse engineering human neurodegenerative disease using pluripotent stem cell technology

Stem Cells for Neuro-regeneration: State of the Art

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