Use of differentiated pluripotent stem cells in replacement therapy for treating disease

Fox, Ira J.; Daley, George Q.; Goldman, Steven A.; Huard, Johnny; Kamp, Timothy J.; Trucco, Massimo

doi:10.1126/science.1247391

Cited by 254 publications

(175 citation statements)

References 148 publications

(134 reference statements)

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“…As a result, OPCs -also referred to in the literature synonymously as glial progenitor cells -have become of great interest as potential vectors for the restoration of myelin in the demyelinated brain and spinal cord. In particular, the generation of myelinogenic OPCs from human pluripotent stem cells (hPSCs) might provide a common cellular reagent by which to treat the entire range of demyelinating disorders (Fox et al, 2014) (Fig. 1) On that basis, in this Primer we will address recent insights into OPC and oligodendrocyte development, how these data have guided the development of techniques by which to generate OPCs and oligodendrocytes from pluripotent cells, and how the cells thereby generated might be reasonably employed as clinical therapeutics.…”

Section: Introductionmentioning

confidence: 99%

How to make an oligodendrocyte

2015

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Oligodendrocytes produce myelin, an insulating sheath required for the saltatory conduction of electrical impulses along axons. Oligodendrocyte loss results in demyelination, which leads to impaired neurological function in a broad array of diseases ranging from pediatric leukodystrophies and cerebral palsy, to multiple sclerosis and white matter stroke. Accordingly, replacing lost oligodendrocytes, whether by transplanting oligodendrocyte progenitor cells (OPCs) or by mobilizing endogenous progenitors, holds great promise as a therapeutic strategy for the diseases of central white matter. In this Primer, we describe the molecular events regulating oligodendrocyte development and how our understanding of this process has led to the establishment of methods for producing OPCs and oligodendrocytes from embryonic stem cells and induced pluripotent stem cells, as well as directly from somatic cells. In addition, we will discuss the safety of engrafted stem cell-derived OPCs, as well as approaches by which to modulate their differentiation and myelinogenesis in vivo following transplantation.

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Section: Introductionmentioning

confidence: 99%

How to make an oligodendrocyte

2015

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“…Other candidate stem cells include HSCs [33] , BM-MSC [34] , and induced pluripotent stem cells (iPSC) [35] . Because iPSC technology enables autologous transplantation allowing immune compatibility with a host immune system (Figure 2), iPSCs are proposed for replacement therapy in certain diseases [36] . However, potential immune rejection of these autologous iPSCs remains to be tested in clinical trials [37] .…”

Section: Emerging Therapiesmentioning

confidence: 99%

Training stem cells for treatment of malignant brain tumors

Li¹,

Kabeer²,

Vu³

et al. 2014

WJSC

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The treatment of malignant brain tumors remains a challenge. Stem cell technology has been applied in the treatment of brain tumors largely because of the ability of some stem cells to infiltrate into regions within the brain where tumor cells migrate as shown in preclinical studies. However, not all of these efforts can translate in the effective treatment that improves the quality of life for patients. Here, we perform a literature review to identify the problems in the field. Given the lack of efficacy of most stem cell-based agents used in the treatment of malignant brain tumors, we found that stem cell distribution (i.e. , only a fraction of stem cells applied capable of targeting tumors) are among the limiting factors. We provide guidelines for potential improvements in stem cell distribution. Specifically, we use an engineered tissue graft platform that replicates the in vivo microenvironment, and provide our data to validate that this culture platform is viable for producing stem cells that have better stem cell distribution than with the Petri dish culture system.

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“…Recently, cell-based biomaterials have been found to have therapeutic applications for tissue engineering [1][2][3]. Epithelial cells form sheet-like complexes in vivo with an underlying thin layer of extracellular matrix (ECM), known as the basement membrane (BM).…”

Section: Introductionmentioning

confidence: 99%

Active Peptide-Conjugated Chitosan Matrices as an Artificial Basement Membrane

et al. 2015

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Abstract:The basement membrane, a thin extracellular matrix, plays a critical role in tissue development and repair. Laminins are the major component of basement membrane and have diverse biological activities. We have identified various cell-adhesive peptides from laminins and their specific cell surface receptors. Polysaccharides, including chitosan, have been used as scaffolds, which regulate cellular functions for tissue engineering. We have developed laminin-derived active peptide-chitosan matrices as functional scaffolds. The biological activity of the peptides was enhanced when the peptides were conjugated to a chitosan matrix, suggesting that the peptide-chitosan matrix approach has an advantage for an active biomaterial. Further, the laminin peptide-chitosan matrices have the potential to mimic the basement membrane and are useful for tissue engineering as an artificial basement membrane.

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Use of differentiated pluripotent stem cells in replacement therapy for treating disease

Cited by 254 publications

References 148 publications

How to make an oligodendrocyte

How to make an oligodendrocyte

Training stem cells for treatment of malignant brain tumors

Active Peptide-Conjugated Chitosan Matrices as an Artificial Basement Membrane

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