Potential of Induced Pluripotent Stem Cells for Use in Gene Therapy: History, Molecular Bases, and Medical Perspectives
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...
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MDPI AG
2021-05-01
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| Series: | Biomolecules |
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| Online Access: | https://www.mdpi.com/2218-273X/11/5/699 |
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| author | Agnieszka Fus-Kujawa Barbara Mendrek Anna Trybus Karolina Bajdak-Rusinek Karolina L. Stepien Aleksander L. Sieron |
| author_facet | Agnieszka Fus-Kujawa Barbara Mendrek Anna Trybus Karolina Bajdak-Rusinek Karolina L. Stepien Aleksander L. Sieron |
| author_sort | Agnieszka Fus-Kujawa |
| collection | DOAJ |
| description | 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. |
| first_indexed | 2024-03-10T11:37:50Z |
| format | Article |
| id | doaj.art-bb21c64fb5a640aea7d2e2e96b788170 |
| institution | Directory Open Access Journal |
| issn | 2218-273X |
| language | English |
| last_indexed | 2024-03-10T11:37:50Z |
| publishDate | 2021-05-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Biomolecules |
| spelling | doaj.art-bb21c64fb5a640aea7d2e2e96b7881702023-11-21T18:43:53ZengMDPI AGBiomolecules2218-273X2021-05-0111569910.3390/biom11050699Potential of Induced Pluripotent Stem Cells for Use in Gene Therapy: History, Molecular Bases, and Medical PerspectivesAgnieszka Fus-Kujawa0Barbara Mendrek1Anna Trybus2Karolina Bajdak-Rusinek3Karolina L. Stepien4Aleksander L. Sieron5Department of Molecular Biology, Faculty of Medical Sciences in Katowice, Medical University of Silesia, Katowice, Medykow 18 Street, 40-752 Katowice, PolandCentre of Polymer and Carbon Materials, Polish Academy of Sciences, M. Curie-Sklodowskiej 34, 41-819 Zabrze, PolandDepartment of Molecular Biology, Faculty of Medical Sciences in Katowice, Medical University of Silesia, Katowice, Medykow 18 Street, 40-752 Katowice, PolandDepartment of Medical Genetics, Faculty of Medical Sciences in Katowice, Medical University of Silesia, Katowice, Medykow 18 Street, 40-752 Katowice, PolandDepartment of Molecular Biology, Faculty of Medical Sciences in Katowice, Medical University of Silesia, Katowice, Medykow 18 Street, 40-752 Katowice, PolandDepartment of Molecular Biology, Faculty of Medical Sciences in Katowice, Medical University of Silesia, Katowice, Medykow 18 Street, 40-752 Katowice, PolandInduced 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.https://www.mdpi.com/2218-273X/11/5/699iPSCsstem cellsgene therapydifferentiationauto-transplantationregenerative medicine |
| spellingShingle | Agnieszka Fus-Kujawa Barbara Mendrek Anna Trybus Karolina Bajdak-Rusinek Karolina L. Stepien Aleksander L. Sieron Potential of Induced Pluripotent Stem Cells for Use in Gene Therapy: History, Molecular Bases, and Medical Perspectives Biomolecules iPSCs stem cells gene therapy differentiation auto-transplantation regenerative medicine |
| title | Potential of Induced Pluripotent Stem Cells for Use in Gene Therapy: History, Molecular Bases, and Medical Perspectives |
| title_full | Potential of Induced Pluripotent Stem Cells for Use in Gene Therapy: History, Molecular Bases, and Medical Perspectives |
| title_fullStr | Potential of Induced Pluripotent Stem Cells for Use in Gene Therapy: History, Molecular Bases, and Medical Perspectives |
| title_full_unstemmed | Potential of Induced Pluripotent Stem Cells for Use in Gene Therapy: History, Molecular Bases, and Medical Perspectives |
| title_short | Potential of Induced Pluripotent Stem Cells for Use in Gene Therapy: History, Molecular Bases, and Medical Perspectives |
| title_sort | potential of induced pluripotent stem cells for use in gene therapy history molecular bases and medical perspectives |
| topic | iPSCs stem cells gene therapy differentiation auto-transplantation regenerative medicine |
| url | https://www.mdpi.com/2218-273X/11/5/699 |
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