The potential of induced pluripotent stem cells in models of neurological disorders: implications on future therapy

Crook, Jeremy Micah; Wallace, Gordon G.; Tomaskovic-Crook, Eva

doi:10.1586/14737175.2015.1013096

Cited by 12 publications

(4 citation statements)

References 133 publications

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“…Ethical concerns and the fear of rejection when using allogeneic cells have prevented clinical trials using ES cells, but the introduction of iPS cells has spurred research into the personalized use of pluripotent cells as a therapy for neurodegenerative disorders (Crook et al , 2015; Inoue et al , 2014). These induced pluripotent cells can differentiate into cell types of the three germ layers.…”

Section: Stem Cells Are Pluripotent/multipotent Cells Capable Of Tissmentioning

confidence: 99%

Stem cell transplantation therapy for multifaceted therapeutic benefits after stroke

Wei

Jiang

et al. 2017

Progress in Neurobiology

127

117

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One of the exciting advances in modern medicine and life science is cell-based neurovascular regeneration of damaged brain tissues and repair of neuronal structures. The progress in stem cell biology and creation of adult induced pluripotent stem (iPS) cells has significantly improved basic and pre-clinical research in disease mechanisms and generated enthusiasm for potential applications in the treatment of central nervous system (CNS) diseases including stroke. Endogenous neural stem cells and cultured stem cells are capable of self-renewal and give rise to virtually all types of cells essential for the makeup of neuronal structures. Meanwhile, stem cells and neural progenitor cells are well-known for their potential for trophic support after transplantation into the ischemic brain. Thus, stem cell-based therapies provide an attractive future for protecting and repairing damaged brain tissues after injury and in various disease states. Moreover, basic research on naïve and differentiated stem cells including iPS cells has markedly improved our understanding of cellular and molecular mechanisms of neurological disorders, and provides a platform for the discovery of novel drug targets. The latest advances indicate that combinatorial approaches using cell based therapy with additional treatments such as protective reagents, preconditioning strategies and rehabilitation therapy can significantly improve therapeutic benefits. In this review, we will discuss the characteristics of cell therapy in different ischemic models and the application of stem cells and progenitor cells as regenerative medicine for the treatment of stroke.

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Section: Stem Cells Are Pluripotent/multipotent Cells Capable Of Tissmentioning

confidence: 99%

Stem cell transplantation therapy for multifaceted therapeutic benefits after stroke

Wei

Jiang

et al. 2017

Progress in Neurobiology

127

117

View full text Add to dashboard Cite

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“…Several reviews are available that discuss the importance of rigor and reproducibility of iPS neurons for targeted drug screens. [36][37][38][39] Essentially, NPC derivation protocols should meet exacting requirements for reproducibility, consistent yields of NPC and iPS neurons, scalability for highthroughput and content screens, and cost/time effectiveness. 36 The two-dimensional monolayer of NPC induction appears to be more aligned with the needs of high-throughput/content applications.…”

Section: Commericialized Kits: a Way To Cut Down On Lot-to-lot Variabmentioning

confidence: 99%

Neural Progenitor Cell Derivation Methodologies for Drug Discovery Applications

Porterfield

2020

ASSAY and Drug Development Technologies

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Inducible pluripotent stem cells (iPSCs) are being used to model brain disorders across the continuum of neurodevelopment, neurodegenerative, and neuropsychiatric disease allowing for the mechanistic unraveling of the neurological disease state. Subsequently, there is a diverse array of cell model systems that can be used for target validation, pharmacodynamic endpoint development, and high-throughput/content assay development and screening. However, to successfully model neurological disorders with iPSCs, the disease-relevant neuron must be first identified, and it is critical to have the appropriate neuronal progenitor cell derivation and neuron differentiation protocols available to produce desired neuronal phenotypes. Moreover, special considerations are necessary if adaptation to high-throughput/content assay systems is anticipated. Discussed here are the three-dimensional embryoid bodyneural rosette and two-dimensional monolayer methodologies to derive iPS neural progenitor cells and neurons with a specific focus on cortical neurons. Outlined are some of the commonalities, advantages, and disadvantages associated with both methodologies.

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“…ADSCs also have the ability to regulate the inflammatory response and hold antioxidant capacity 12 . Moreover, under the induction of specific genetic signals, ADSCs can differentiate into neuron-like or glia-like cells 13,14 . However, ADSCs are difficult to transfect 14 , thus driving the design of better gene delivery systems.…”

Section: Introductionmentioning

confidence: 99%

Highly branched poly(β-amino ester) delivery of minicircle DNA for transfection of neurodegenerative disease related cells

et al. 2019

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Current therapies for most neurodegenerative disorders are only symptomatic in nature and do not change the course of the disease. Gene therapy plays an important role in disease modifying therapeutic strategies. Herein, we have designed and optimized a series of highly branched poly(β-amino ester)s (HPAEs) containing biodegradable disulfide units in the HPAE backbone (HPAESS) and guanidine moieties (HPAESG) at the extremities. The optimized polymers are used to deliver minicircle DNA to multipotent adipose derived stem cells (ADSCs) and astrocytes, and high transfection efficiency is achieved (77% in human ADSCs and 52% in primary astrocytes) whilst preserving over 90% cell viability. Furthermore, the top-performing candidate mediates high levels of nerve growth factor (NGF) secretion from astrocytes, causing neurite outgrowth from a model neuron cell line. This synergistic gene delivery system provides a viable method for highly efficient non-viral transfection of ADSCs and astrocytes.

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The potential of induced pluripotent stem cells in models of neurological disorders: implications on future therapy

Cited by 12 publications

References 133 publications

Stem cell transplantation therapy for multifaceted therapeutic benefits after stroke

Stem cell transplantation therapy for multifaceted therapeutic benefits after stroke

Neural Progenitor Cell Derivation Methodologies for Drug Discovery Applications

Highly branched poly(β-amino ester) delivery of minicircle DNA for transfection of neurodegenerative disease related cells

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