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      Challenges in the clinical application of induced pluripotent stem cells

      research-article
      1 ,
      Stem Cell Research & Therapy
      BioMed Central

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          Abstract

          The advent of human induced pluripotent stem cells has been heralded as a major breakthrough in the study of pluripotent stem cells, for these cells have yielded fundamental insights into the reprogrammability of somatic cell fates, but also because of their seemingly great promise in applications, including potential uses in cell therapy. Several recent reports in the scientific literature and mass media, however, have challenged this concept for reasons of biological function and business feasibility, presenting an important opportunity to re-examine the prospects for human induced pluripotent stem cells in medicine. In this commentary, I will outline a number of hurdles that will need to be cleared if these cells are to fulfil their clinical promise, and suggest avenues that might facilitate these important endeavours.

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          Most cited references7

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          Technical challenges in using human induced pluripotent stem cells to model disease.

          Reprogramming of human somatic cells uses readily accessible tissue, such as skin or blood, to generate embryonic-like induced pluripotent stem cells (iPSCs). This procedure has been applied to somatic cells from patients who are classified into a disease group, thus creating "disease-specific" iPSCs. Here, we examine the challenges and assumptions in creating a disease model from a single cell of the patient. Both the kinetics of disease onset and progression as well as the spatial localization of disease in the patient's body are challenges to disease modeling. New tools in genetic modification, reprogramming, biomaterials, and animal models can be used for addressing these challenges.
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            Progress toward the clinical application of patient-specific pluripotent stem cells.

            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.
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              Prospects for stem cell-based therapy.

              Resident pools of somatic stem cells in many organs are responsible for tissue maintenance and repair. The goal of regenerative medicine is to exploit these cells either by transplanting them from an exogenous source or by activating endogenous stem cells pharmacologically. For diseases caused by mutations in a single gene, the therapeutic goal is tissue replacement using stem cells engineered to correct the genetic defect. However, a number of technical hurdles must be overcome before therapies based on pluripotent human stem cells can enter the clinic.
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                Author and article information

                Journal
                Stem Cell Res Ther
                Stem Cell Research & Therapy
                BioMed Central
                1757-6512
                2010
                12 April 2010
                : 1
                : 1
                : 9
                Affiliations
                [1 ]Science Policy and Ethics Unit, RIKEN Center for Developmental Biology, 2-2-3 Minatojima Minamimachi, Chuo-ku, Kobe, Japan 650-0047
                Article
                scrt9
                10.1186/scrt9
                3226303
                20504290
                44d8fdb5-5856-4a6f-a91d-3709a5477956
                Copyright ©2010 BioMed Central Ltd
                History
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                Commentary

                Molecular medicine
                Molecular medicine

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