scholarly journals Potential of Induced Pluripotent Stem Cells for Use in Gene Therapy: History, Molecular Bases, and Medical Perspectives

Biomolecules ◽  
2021 ◽  
Vol 11 (5) ◽  
pp. 699
Author(s):  
Agnieszka Fus-Kujawa ◽  
Barbara Mendrek ◽  
Anna Trybus ◽  
Karolina Bajdak-Rusinek ◽  
Karolina L. Stepien ◽  
...  
Keyword(s):  
Stem Cells ◽  
Gene Therapy ◽  
Induced Pluripotent Stem Cells ◽  
Pluripotent Stem Cells ◽  
Genetic Diseases ◽  
Embryonic Stem ◽  
Patient Specific ◽  
Clinical Settings ◽  
Induced Pluripotent ◽  
Molecular Bases

Induced pluripotent stem cells (iPSCs) are defined as reprogrammed somatic cells exhibiting embryonic stem cell characteristics. Since their discovery in 2006, efforts have been made to utilize iPSCs in clinical settings. One of the promising fields of medicine, in which genetically patient-specific stem cells may prove themselves useful, is gene therapy. iPSCs technology holds potential in both creating models of genetic diseases and delivering therapeutic agents into the organism via auto-transplants, which reduces the risk of rejection compared to allotransplants. However, in order to safely administer genetically corrected stem cells into patients’ tissues, efforts must be made to establish stably pluripotent stem cells and reduce the risk of insertional tumorigenesis. In order to achieve this, optimal reprogramming factors and vectors must be considered. Therefore, in this review, the molecular bases of reprogramming safe iPSCs for clinical applications and recent attempts to translate iPSCs technology into the clinical setting are discussed.

scholarly journals The Application of Induced Pluripotent Stem Cells in Pathogenesis Study and Gene Therapy for Vascular Disorders: Current Progress and Future Challenges

2019 ◽  
Vol 2019 ◽  
pp. 1-9 ◽  
Author(s):  
Guang-Yin Peng ◽  
Yang Lin ◽  
Jing-Jing Li ◽  
Ying Wang ◽  
Hao-Yue Huang ◽  
...  
Keyword(s):  
Stem Cells ◽  
Gene Therapy ◽  
Induced Pluripotent Stem Cells ◽  
Pluripotent Stem Cells ◽  
Disease Modeling ◽  
Vascular Diseases ◽  
Patient Specific ◽  
High Morbidity ◽  
Vascular Disorders ◽  
Induced Pluripotent

Vascular disorders are complex diseases with high morbidity and mortality. Among them, the dilated macrovascular diseases (MVD), such as aortic aneurysm and aortic dissection, have presented a huge threat to human health. The pathogenesis of vascular diseases is mostly associated with property alteration of vascular endothelial cells (VECs) and vascular smooth muscle cells (VSMCs). Studies have confirmed that induced pluripotent stem cells (iPSCs) can be proliferated and differentiated into other somatic cells, such as VECs and VSMCs. And patient-specific cells could provide detailed human-associated information in regard to pathogenesis or drug responses. In addition, differentiated ECs from iPSC have been widely used in disease modeling as a cell therapy. In this review, we mainly discussed the application of hiPSCs in investigating the pathological mechanism of different inherited vascular diseases and provide a comprehensive understanding of hiPSCs in the field of clinical diagnosis and gene therapy.

Actual strategies in human induced pluripotent stem cells (hiPSCs) differentiation – perspectives and challenges

2016 ◽  
Vol 52 (2) ◽  
pp. 123-136
Author(s):  
Ewelina Augustyniak ◽  
Katarzyna Kulcenty ◽  
Michał Lach ◽  
Igor Piotrowski ◽  
Wiktoria Maria Suchorska
Keyword(s):  
Stem Cells ◽  
Induced Pluripotent Stem Cells ◽  
Pluripotent Stem Cells ◽  
Embryonic Stem ◽  
Patient Specific ◽  
Human Embryos ◽  
Alternative Approaches ◽  
Induced Pluripotent ◽  
Autologous Grafts ◽  
Derivatives Of

The application of stem cells (SCs) in regenerative medicine has recently become a rapidly growing field, holding promise for combating a number of currently incurable disorders: including diabetes, neurodegenerative, retinal and cardiac diseases, as well as muscular dystrophy. The search for alternative approaches led to the development of human induced pluripotent stem cells (hiPSCs) which have unrestricted proliferative activity and pluripotency – the capacity to differentiation into derivatives of three germ layers (meso-, ecto – and endoderm). Because hiPSCs are developed from adult human cells throughout the forced expression of pluripotency factors, they are free from the ethical concerns associated with human embryonic stem cells (hESCs), that creation involves the destruction of human embryos. Moreover, the use of hiPSCs contributes to the development of personalized medicine that exploits patient-specific cells extremely useful in autologous grafts. In the present study the methods of hiPSCs differentiation into stem cell-derived neurons, cardiomyocytes, chondrocytes and osteocytes were summarized and evaluated having regard to their most important aspects.

scholarly journals Generation of induced pluripotent stem cells from human blood

Blood ◽  
2009 ◽  
Vol 113 (22) ◽  
pp. 5476-5479 ◽  
Author(s):  
Yuin-Han Loh ◽  
Suneet Agarwal ◽  
In-Hyun Park ◽  
Achia Urbach ◽  
Hongguang Huo ◽  
...  
Keyword(s):  
Stem Cells ◽  
Transcription Factors ◽  
Induced Pluripotent Stem Cells ◽  
Human Blood ◽  
Pluripotent Stem Cells ◽  
Methylation Status ◽  
Embryonic Stem ◽  
Dermal Fibroblasts ◽  
Patient Specific ◽  
Induced Pluripotent

Human dermal fibroblasts obtained by skin biopsy can be reprogrammed directly to pluripotency by the ectopic expression of defined transcription factors. Here, we describe the derivation of induced pluripotent stem cells from CD34+ mobilized human peripheral blood cells using retroviral transduction of OCT4/SOX2/KLF4/MYC. Blood-derived human induced pluripotent stem cells are indistinguishable from human embryonic stem cells with respect to morphology, expression of surface antigens, and pluripotency-associated transcription factors, DNA methylation status at pluripotent cell-specific genes, and the capacity to differentiate in vitro and in teratomas. The ability to reprogram cells from human blood will allow the generation of patient-specific stem cells for diseases in which the disease-causing somatic mutations are restricted to cells of the hematopoietic lineage.

Culture and Characteristics of Human Induced Pluripotent Stem Cells

2011 ◽  
Vol 268-270 ◽  
pp. 835-837
Author(s):  
De Wu Liu ◽  
Yong Tie Li ◽  
De Ming Liu ◽  
Pu Ning
Keyword(s):  
Stem Cells ◽  
Alkaline Phosphatase ◽  
Embryonic Stem Cells ◽  
Induced Pluripotent Stem Cells ◽  
Human Embryonic Stem Cells ◽  
Pluripotent Stem Cells ◽  
Embryoid Body ◽  
Embryonic Stem ◽  
Patient Specific ◽  
Induced Pluripotent

Human induced pluripotent stem cells is promising for regenerative medicine and tissue engineering. In this chapter, we focus on the culture and characteristics of human induced pluripotent stem cells. The induced pluripotent stem cells were plated on murine embryonic fibroblast feeder cells and expanded in human embryonic stem cells media contained basic fibroblast growth factor. The cells were passaged by collagenase IV digestion method and observed under invert microscope. The expression of alkaline phosphatase was detected by immunocytochemistry. The cultured induced pluripotent stem cells grew well and stability with similar characteristics of human embryonic stem cells. These cells also expressed alkaline phosphatase. They formed embryoid body in feeder-free and suspension culture conditions. The results provide an experimental basis for improvement of induction study and further application to generate patient-specific induced pluripotent stem cells.

scholarly journals Ten years of progress and promise of induced pluripotent stem cells: historical origins, characteristics, mechanisms, limitations, and potential applications

2017 ◽  
Author(s):  
Adekunle Ebenezer Omole ◽  
Adegbenro Omotuyi John Fakoya
Keyword(s):  
Stem Cells ◽  
Stem Cell ◽  
Transcription Factors ◽  
Induced Pluripotent Stem Cells ◽  
Molecular Mechanism ◽  
Pluripotent Stem Cells ◽  
Ectopic Expression ◽  
Embryonic Stem ◽  
Patient Specific ◽  
Induced Pluripotent

The discovery of induced pluripotent stem cells (iPSCs) by Shinya Yamanaka in 2006 was heralded as a major breakthrough of the decade in stem cell research. The ability to reprogrammed human somatic cells to a pluripotent embryonic stem cell-like state through the ectopic expression of a combination of embryonic transcription factors was greeted with great excitement by scientists and bioethicists. The reprogramming technology offers the opportunity to generate patient-specific stem cells for modeling human diseases, drug development and screening, and individualized regenerative cell therapy. However, fundamental questions have been raised regarding the molecular mechanism of iPSCs generation, a process still poorly understood by scientists. The efficiency of reprogramming of iPSCs remains low due to the effect of various barriers of reprogramming. There is also the risk of chromosomal instability and oncogenic transformation associated with the use of viral vectors, such as retrovirus and lentivirus, which deliver the reprogramming transcription factors by integration in the host cell genome. These challenges can hinder the therapeutic prospects and promise of iPSCs and their clinical applications. Consequently, extensive studies have been done to elucidate the molecular mechanism of reprogramming and novel strategies have been identified which help to improve the efficiency of reprogramming methods and overcome the safety concerns linked with iPSCs generation. Distinct barriers and enhancers of reprogramming have been elucidated and non-integrating reprogramming methods have been reported. Here, we summarize the progress and the recent advances that have been made over the last 10 years in the iPSCs field, with emphasis on the molecular mechanism of reprogramming, strategies to improve the efficiency of reprogramming, characteristics and limitations of iPSCs, and the progress made in the applications of iPSCs in the field of disease modelling, drug discovery and regenerative medicine. Additionally, this study appraised the role of genomic editing technology in the generation of healthy iPSCs.

scholarly journals Ten years of progress and promise of induced pluripotent stem cells: historical origins, characteristics, mechanisms, limitations, and potential applications

2017 ◽  
Author(s):  
Adekunle Ebenezer Omole ◽  
Adegbenro Omotuyi John Fakoya
Keyword(s):  
Stem Cells ◽  
Stem Cell ◽  
Transcription Factors ◽  
Induced Pluripotent Stem Cells ◽  
Molecular Mechanism ◽  
Pluripotent Stem Cells ◽  
Ectopic Expression ◽  
Embryonic Stem ◽  
Patient Specific ◽  
Induced Pluripotent

The discovery of induced pluripotent stem cells (iPSCs) by Shinya Yamanaka in 2006 was heralded as a major breakthrough of the decade in stem cell research. The ability to reprogrammed human somatic cells to a pluripotent embryonic stem cell-like state through the ectopic expression of a combination of embryonic transcription factors was greeted with great excitement by scientists and bioethicists. The reprogramming technology offers the opportunity to generate patient-specific stem cells for modeling human diseases, drug development and screening, and individualized regenerative cell therapy. However, fundamental questions have been raised regarding the molecular mechanism of iPSCs generation, a process still poorly understood by scientists. The efficiency of reprogramming of iPSCs remains low due to the effect of various barriers of reprogramming. There is also the risk of chromosomal instability and oncogenic transformation associated with the use of viral vectors, such as retrovirus and lentivirus, which deliver the reprogramming transcription factors by integration in the host cell genome. These challenges can hinder the therapeutic prospects and promise of iPSCs and their clinical applications. Consequently, extensive studies have been done to elucidate the molecular mechanism of reprogramming and novel strategies have been identified which help to improve the efficiency of reprogramming methods and overcome the safety concerns linked with iPSCs generation. Distinct barriers and enhancers of reprogramming have been elucidated and non-integrating reprogramming methods have been reported. Here, we summarize the progress and the recent advances that have been made over the last 10 years in the iPSCs field, with emphasis on the molecular mechanism of reprogramming, strategies to improve the efficiency of reprogramming, characteristics and limitations of iPSCs, and the progress made in the applications of iPSCs in the field of disease modelling, drug discovery and regenerative medicine. Additionally, this study appraised the role of genomic editing technology in the generation of healthy iPSCs.

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