Ten Years of iPSCs: Current Status and Future Perspectives

A special issue of Cells (ISSN 2073-4409).

Deadline for manuscript submissions: closed (30 November 2017) | Viewed by 67291

Special Issue Editor


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Guest Editor
Chief Executive Officer, Enthera, Milano, Italy
Interests: induced pluripotent stem cells; epigenetics; hematopoiesis; neurogenesis; early and late stage clinical trials
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Special Issue Information

Dear Colleagues,

The landmark finding in 2006, that lineage-restricted cells could be reprogrammed to a pluripotent state, has revolutionized the regenerative medicine field, opening new therapeutic prospects for several severe conditions. Induced pluripotent Stem Cells (iPS) can be obtained by using numerous reprogramming strategies that became step-by-step safer by avoiding the use of oncogenes and the integration of transgenes.

Pluripotent stem cells hold the greatest promise given their potential to differentiate into all the cell types of the human body and their potential as treatment of several genetic or degenerative diseases.

In this Special Issue, we invite your contributions, either in the form of original research articles, reviews, or shorter “Perspective” articles on all aspects related to the theme of “Transforming Induced Pluripotent Stem Cells into Novel Therapeutic Opportunities”. Articles with mechanistic and functional insights from a cell and molecular biological perspective are especially welcome.

Dr. Giovanni Amabile
Guest Editor

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Keywords

  • induced pluripotent stem cells
  • reprogramming
  • regenerative medicine
  • cell therapies
  • differentiation
  • epigenetics
  • clinical development

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Published Papers (6 papers)

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Review

31 pages, 1295 KiB  
Review
Human-Induced Pluripotent Stem Cell Technology and Cardiomyocyte Generation: Progress and Clinical Applications
by Angela Di Baldassarre, Elisa Cimetta, Sveva Bollini, Giulia Gaggi and Barbara Ghinassi
Cells 2018, 7(6), 48; https://doi.org/10.3390/cells7060048 - 25 May 2018
Cited by 50 | Viewed by 7886
Abstract
Human-induced pluripotent stem cells (hiPSCs) are reprogrammed cells that have hallmarks similar to embryonic stem cells including the capacity of self-renewal and differentiation into cardiac myocytes. The improvements in reprogramming and differentiating methods achieved in the past 10 years widened the use of [...] Read more.
Human-induced pluripotent stem cells (hiPSCs) are reprogrammed cells that have hallmarks similar to embryonic stem cells including the capacity of self-renewal and differentiation into cardiac myocytes. The improvements in reprogramming and differentiating methods achieved in the past 10 years widened the use of hiPSCs, especially in cardiac research. hiPSC-derived cardiac myocytes (CMs) recapitulate phenotypic differences caused by genetic variations, making them attractive human disease models and useful tools for drug discovery and toxicology testing. In addition, hiPSCs can be used as sources of cells for cardiac regeneration in animal models. Here, we review the advances in the genetic and epigenetic control of cardiomyogenesis that underlies the significant improvement of the induced reprogramming of somatic cells to CMs; the methods used to improve scalability of throughput assays for functional screening and drug testing in vitro; the phenotypic characteristics of hiPSCs-derived CMs and their ability to rescue injured CMs through paracrine effects; we also cover the novel approaches in tissue engineering for hiPSC-derived cardiac tissue generation, and finally, their immunological features and the potential use in biomedical applications. Full article
(This article belongs to the Special Issue Ten Years of iPSCs: Current Status and Future Perspectives)
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646 KiB  
Review
Induced Pluripotent Stem Cell-Derived Red Blood Cells and Platelet Concentrates: From Bench to Bedside
by Daniele Focosi and Giovanni Amabile
Cells 2018, 7(1), 2; https://doi.org/10.3390/cells7010002 - 27 Dec 2017
Cited by 22 | Viewed by 7084
Abstract
Red blood cells and platelets are anucleate blood components indispensable for oxygen delivery and hemostasis, respectively. Derivation of these blood elements from induced pluripotent stem (iPS) cells has the potential to develop blood donor-independent and genetic manipulation-prone products to complement or replace current [...] Read more.
Red blood cells and platelets are anucleate blood components indispensable for oxygen delivery and hemostasis, respectively. Derivation of these blood elements from induced pluripotent stem (iPS) cells has the potential to develop blood donor-independent and genetic manipulation-prone products to complement or replace current transfusion banking, also minimizing the risk of alloimmunization. While the production of erythrocytes from iPS cells has challenges to overcome, such as differentiation into adult-type phenotype that functions properly after transfusion, platelet products are qualitatively and quantitatively approaching a clinically-applicable level owing to advances in expandable megakaryocyte (MK) lines, platelet-producing bioreactors, and novel reagents. Guidelines that assure the quality of iPS cells-derived blood products for clinical application represent a novel challenge for regulatory agencies. Considering the minimal risk of tumorigenicity and the expected significant demand of such products, ex vivo production of iPS-derived blood components can pave the way for iPS translation into the clinic. Full article
(This article belongs to the Special Issue Ten Years of iPSCs: Current Status and Future Perspectives)
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1213 KiB  
Review
Application of Induced Pluripotent Stem Cell Technology to the Study of Hematological Diseases
by Mailin Li, Pasquale Cascino, Simone Ummarino and Annalisa Di Ruscio
Cells 2017, 6(1), 7; https://doi.org/10.3390/cells6010007 - 8 Mar 2017
Cited by 12 | Viewed by 15836
Abstract
The burst of reprogramming technology in recent years has revolutionized the field of stem cell biology, offering new opportunities for personalized, regenerative therapies. The direct reprogramming of somatic cells to induced pluripotent stem cells (iPSCs) has provided an invaluable tool to study and [...] Read more.
The burst of reprogramming technology in recent years has revolutionized the field of stem cell biology, offering new opportunities for personalized, regenerative therapies. The direct reprogramming of somatic cells to induced pluripotent stem cells (iPSCs) has provided an invaluable tool to study and model a wide range of human diseases. Here, we review the transforming potential of such a strategy in research and in therapies applicable to the hematology field. Full article
(This article belongs to the Special Issue Ten Years of iPSCs: Current Status and Future Perspectives)
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655 KiB  
Review
May I Cut in? Gene Editing Approaches in Human Induced Pluripotent Stem Cells
by Nicholas Brookhouser, Sreedevi Raman, Christopher Potts and David. A. Brafman
Cells 2017, 6(1), 5; https://doi.org/10.3390/cells6010005 - 6 Feb 2017
Cited by 37 | Viewed by 18751
Abstract
In the decade since Yamanaka and colleagues described methods to reprogram somatic cells into a pluripotent state, human induced pluripotent stem cells (hiPSCs) have demonstrated tremendous promise in numerous disease modeling, drug discovery, and regenerative medicine applications. More recently, the development and refinement [...] Read more.
In the decade since Yamanaka and colleagues described methods to reprogram somatic cells into a pluripotent state, human induced pluripotent stem cells (hiPSCs) have demonstrated tremendous promise in numerous disease modeling, drug discovery, and regenerative medicine applications. More recently, the development and refinement of advanced gene transduction and editing technologies have further accelerated the potential of hiPSCs. In this review, we discuss the various gene editing technologies that are being implemented with hiPSCs. Specifically, we describe the emergence of technologies including zinc-finger nuclease (ZFN), transcription activator-like effector nuclease (TALEN), and clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9 that can be used to edit the genome at precise locations, and discuss the strengths and weaknesses of each of these technologies. In addition, we present the current applications of these technologies in elucidating the mechanisms of human development and disease, developing novel and effective therapeutic molecules, and engineering cell-based therapies. Finally, we discuss the emerging technological advances in targeted gene editing methods. Full article
(This article belongs to the Special Issue Ten Years of iPSCs: Current Status and Future Perspectives)
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1061 KiB  
Review
Translational Prospects and Challenges in Human Induced Pluripotent Stem Cell Research in Drug Discovery
by Masaki Hosoya and Katherine Czysz
Cells 2016, 5(4), 46; https://doi.org/10.3390/cells5040046 - 21 Dec 2016
Cited by 22 | Viewed by 7782
Abstract
Despite continuous efforts to improve the process of drug discovery and development, achieving success at the clinical stage remains challenging because of a persistent translational gap between the preclinical and clinical settings. Under these circumstances, the discovery of human induced pluripotent stem (iPS) [...] Read more.
Despite continuous efforts to improve the process of drug discovery and development, achieving success at the clinical stage remains challenging because of a persistent translational gap between the preclinical and clinical settings. Under these circumstances, the discovery of human induced pluripotent stem (iPS) cells has brought new hope to the drug discovery field because they enable scientists to humanize a variety of pharmacological and toxicological models in vitro. The availability of human iPS cell-derived cells, particularly as an alternative for difficult-to-access tissues and organs, is increasing steadily; however, their use in the field of translational medicine remains challenging. Biomarkers are an essential part of the translational effort to shift new discoveries from bench to bedside as they provide a measurable indicator with which to evaluate pharmacological and toxicological effects in both the preclinical and clinical settings. In general, during the preclinical stage of the drug development process, in vitro models that are established to recapitulate human diseases are validated by using a set of biomarkers; however, their translatability to a clinical setting remains problematic. This review provides an overview of current strategies for human iPS cell-based drug discovery from the perspective of translational research, and discusses the importance of early consideration of clinically relevant biomarkers. Full article
(This article belongs to the Special Issue Ten Years of iPSCs: Current Status and Future Perspectives)
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911 KiB  
Review
Potential of Induced Pluripotent Stem Cells (iPSCs) for Treating Age-Related Macular Degeneration (AMD)
by Mark Fields, Hui Cai, Jie Gong and Lucian Del Priore
Cells 2016, 5(4), 44; https://doi.org/10.3390/cells5040044 - 8 Dec 2016
Cited by 27 | Viewed by 8641
Abstract
The field of stem cell biology has rapidly evolved in the last few decades. In the area of regenerative medicine, clinical applications using stem cells hold the potential to be a powerful tool in the treatment of a wide variety of diseases, in [...] Read more.
The field of stem cell biology has rapidly evolved in the last few decades. In the area of regenerative medicine, clinical applications using stem cells hold the potential to be a powerful tool in the treatment of a wide variety of diseases, in particular, disorders of the eye. Embryonic stem cells (ESCs) and induced pluripotent stem cells (iPSCs) are promising technologies that can potentially provide an unlimited source of cells for cell replacement therapy in the treatment of retinal degenerative disorders such as age-related macular degeneration (AMD), Stargardt disease, and other disorders. ESCs and iPSCs have been used to generate retinal pigment epithelium (RPE) cells and their functional behavior has been tested in vitro and in vivo in animal models. Additionally, iPSC-derived RPE cells provide an autologous source of cells for therapeutic use, as well as allow for novel approaches in disease modeling and drug development platforms. Clinical trials are currently testing the safety and efficacy of these cells in patients with AMD. In this review, the current status of iPSC disease modeling of AMD is discussed, as well as the challenges and potential of this technology as a viable option for cell replacement therapy in retinal degeneration. Full article
(This article belongs to the Special Issue Ten Years of iPSCs: Current Status and Future Perspectives)
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