Human embryonic stem cells as a cellular model for human disorders

Ben-Nun, Inbar Friedrich; Benvenisty, Nissim

doi:10.1016/j.mce.2006.03.034

Cited by 38 publications

(24 citation statements)

References 35 publications

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“…The concept of utilizing ES and now iPS cells to model a disease in a culture dish is based on the unique capacity of these cells to continuously self-renew and their potential to give rise to all cell types in the human body (71,88). Thus, pluripotent cells could provide a limitless reservoir of cell types that in many cases were not previously possible to obtain, for example, the motor and dopaminergic neurons affected in ALS and PD.…”

Section: Figurementioning

confidence: 99%

Progress toward the clinical application of patient-specific pluripotent stem cells

Kiskinis¹,

Eggan²

2010

J. Clin. Invest.

323

249

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Induced pluripotent stem (iPS) cells are generated by epigenetic reprogramming of somatic cells through the exogenous expression of transcription factors. These cells, just like embryonic stem cells, are likely to have a major impact on regenerative medicine, because they self-renew and retain the potential to be differentiated into all cell types of the human body. In this Review, we describe the current state of iPS cell technology, including approaches by which they are generated and what is known about their biology, and discuss the potential applications of these cells for disease modeling, drug discovery, and, eventually, cell replacement therapy. IntroductionHuman ES cells, which are derived from the inner cell mass of blastocyst stage embryos, have the unique ability to self-renew indefinitely while maintaining the potential to give rise to all cell types in the human body (1). Induced pluripotent stem (iPS) cells share these salient characteristics of ES cells but are instead generated via reprogramming of somatic cells through the forced expression of key transcription factors (2). The seminal achievement of induced pluripotency holds great promise for regenerative medicine. Patient-specific iPS cells could provide useful platforms for drug discovery and offer unprecedented insights into disease mechanisms and, in the long term, may be used for cell and tissue replacement therapies.The successful cloning of animals such as Dolly the sheep in 1997 (3, 4) and the subsequent derivation of human ES cells in 1998 (1) brought forward the concept of therapeutic cloning, in which pluripotent ES cell lines tailored to the genetic makeup of specific individuals might provide a plentiful source of therapeutic cells (5). Although significant advancements toward this goal have been made (6, 7), successful somatic cell nuclear transfer (SCNT) (a technique whereby the DNA of an unfertilized egg is replaced by the DNA of a somatic cell) with human cells remains elusive and is fraught with social and logistical concerns. Alternative methods for deriving pluripotent cells, such as cell fusion (8) and culture-induced reprogramming (9), have been developed, but these approaches still suffer from severe practical and technical limitations. In contrast, the generation of pluripotent cells by exogenous expression of transcription factors circumvents many previous limitations, as this approach is not technically demanding and does not require embryonic material or oocytes. We therefore believe that iPS cell technology will have a significant impact on regenerative medicine, and in this article we review current methodologies used for generating iPS cells and then discuss their potential clinical applications.

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Section: Figurementioning

confidence: 99%

Progress toward the clinical application of patient-specific pluripotent stem cells

Kiskinis¹,

Eggan²

2010

J. Clin. Invest.

323

249

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“…Other advantages of stem cells include; the availability of cell types which were not previously available and the ability to investigate cellular renewal, regeneration, expansion as well as differentiation [82]. Stem cells can also be genetically modified using reporter gene construct, thereby providing specific disease models [83].…”

Section: Stem Cell Technologiesmentioning

confidence: 99%

Pre-Clinical Assessment of the Potential Intrinsic Hepatotoxicity of Candidate Drugs

Tonder¹,

Steenkamp²,

Gulumian³

2013

New Insights Into Toxicity and Drug Testing

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“…The usefulness of iPS cells to model a disease in a culture dish is based on the unique capacity of these cells to continuously self-renew and their potential to give rise to all cell types in the human body [Murry CE and Keller G 2008;Friedrich Ben-Nun I, Benveniscy N 2006]. The potential use of iPSCs as treatments for various disorders has been proposed and tested on in vitro and/or in vivo animal models, with promising results.…”

Section: Potential Medical Application Of Ipscsmentioning

confidence: 99%