Induced Pluripotent Stem Cells: Hope in the Treatment of Diseases, including Muscular Dystrophies
Induced pluripotent stem (iPS) cells are laboratory-produced cells that combine the biological advantages of somatic adult and stem cells for cell-based therapy. The reprogramming of cells, such as fibroblasts, to an embryonic stem cell-like state is done by the ectopic expression of transcription f...
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| Format: | Article |
| Language: | English |
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MDPI AG
2020-07-01
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| Series: | International Journal of Molecular Sciences |
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| Online Access: | https://www.mdpi.com/1422-0067/21/15/5467 |
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| author | Daniela Gois Beghini Samuel Iwao Horita Cynthia Machado Cascabulho Luiz Anastácio Alves Andrea Henriques-Pons |
| author_facet | Daniela Gois Beghini Samuel Iwao Horita Cynthia Machado Cascabulho Luiz Anastácio Alves Andrea Henriques-Pons |
| author_sort | Daniela Gois Beghini |
| collection | DOAJ |
| description | Induced pluripotent stem (iPS) cells are laboratory-produced cells that combine the biological advantages of somatic adult and stem cells for cell-based therapy. The reprogramming of cells, such as fibroblasts, to an embryonic stem cell-like state is done by the ectopic expression of transcription factors responsible for generating embryonic stem cell properties. These primary factors are octamer-binding transcription factor 4 (Oct3/4), sex-determining region Y-box 2 (Sox2), Krüppel-like factor 4 (Klf4), and the proto-oncogene protein homolog of avian myelocytomatosis (c-Myc). The somatic cells can be easily obtained from the patient who will be subjected to cellular therapy and be reprogrammed to acquire the necessary high plasticity of embryonic stem cells. These cells have no ethical limitations involved, as in the case of embryonic stem cells, and display minimal immunological rejection risks after transplant. Currently, several clinical trials are in progress, most of them in phase I or II. Still, some inherent risks, such as chromosomal instability, insertional tumors, and teratoma formation, must be overcome to reach full clinical translation. However, with the clinical trials and extensive basic research studying the biology of these cells, a promising future for human cell-based therapies using iPS cells seems to be increasingly clear and close. |
| first_indexed | 2024-03-10T18:05:10Z |
| format | Article |
| id | doaj.art-660f02c712244cc9ab52a11a4429224c |
| institution | Directory Open Access Journal |
| issn | 1661-6596 1422-0067 |
| language | English |
| last_indexed | 2024-03-10T18:05:10Z |
| publishDate | 2020-07-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | International Journal of Molecular Sciences |
| spelling | doaj.art-660f02c712244cc9ab52a11a4429224c2023-11-20T08:34:25ZengMDPI AGInternational Journal of Molecular Sciences1661-65961422-00672020-07-012115546710.3390/ijms21155467Induced Pluripotent Stem Cells: Hope in the Treatment of Diseases, including Muscular DystrophiesDaniela Gois Beghini0Samuel Iwao Horita1Cynthia Machado Cascabulho2Luiz Anastácio Alves3Andrea Henriques-Pons4Laboratório de Inovações em Terapias, Ensino e Bioprodutos, 2- Laboratório de Comunicação Celular, both at the Instituto Oswaldo Cruz, Fundação Oswaldo Cruz, Rio de Janeiro RJ 21040-900, BrazilLaboratório de Inovações em Terapias, Ensino e Bioprodutos, 2- Laboratório de Comunicação Celular, both at the Instituto Oswaldo Cruz, Fundação Oswaldo Cruz, Rio de Janeiro RJ 21040-900, BrazilLaboratório de Inovações em Terapias, Ensino e Bioprodutos, 2- Laboratório de Comunicação Celular, both at the Instituto Oswaldo Cruz, Fundação Oswaldo Cruz, Rio de Janeiro RJ 21040-900, BrazilLaboratório de Inovações em Terapias, Ensino e Bioprodutos, 2- Laboratório de Comunicação Celular, both at the Instituto Oswaldo Cruz, Fundação Oswaldo Cruz, Rio de Janeiro RJ 21040-900, BrazilLaboratório de Inovações em Terapias, Ensino e Bioprodutos, 2- Laboratório de Comunicação Celular, both at the Instituto Oswaldo Cruz, Fundação Oswaldo Cruz, Rio de Janeiro RJ 21040-900, BrazilInduced pluripotent stem (iPS) cells are laboratory-produced cells that combine the biological advantages of somatic adult and stem cells for cell-based therapy. The reprogramming of cells, such as fibroblasts, to an embryonic stem cell-like state is done by the ectopic expression of transcription factors responsible for generating embryonic stem cell properties. These primary factors are octamer-binding transcription factor 4 (Oct3/4), sex-determining region Y-box 2 (Sox2), Krüppel-like factor 4 (Klf4), and the proto-oncogene protein homolog of avian myelocytomatosis (c-Myc). The somatic cells can be easily obtained from the patient who will be subjected to cellular therapy and be reprogrammed to acquire the necessary high plasticity of embryonic stem cells. These cells have no ethical limitations involved, as in the case of embryonic stem cells, and display minimal immunological rejection risks after transplant. Currently, several clinical trials are in progress, most of them in phase I or II. Still, some inherent risks, such as chromosomal instability, insertional tumors, and teratoma formation, must be overcome to reach full clinical translation. However, with the clinical trials and extensive basic research studying the biology of these cells, a promising future for human cell-based therapies using iPS cells seems to be increasingly clear and close.https://www.mdpi.com/1422-0067/21/15/5467induced pluripotent stem cellsregenerationcellular therapystem cellsmuscular dystrophy |
| spellingShingle | Daniela Gois Beghini Samuel Iwao Horita Cynthia Machado Cascabulho Luiz Anastácio Alves Andrea Henriques-Pons Induced Pluripotent Stem Cells: Hope in the Treatment of Diseases, including Muscular Dystrophies International Journal of Molecular Sciences induced pluripotent stem cells regeneration cellular therapy stem cells muscular dystrophy |
| title | Induced Pluripotent Stem Cells: Hope in the Treatment of Diseases, including Muscular Dystrophies |
| title_full | Induced Pluripotent Stem Cells: Hope in the Treatment of Diseases, including Muscular Dystrophies |
| title_fullStr | Induced Pluripotent Stem Cells: Hope in the Treatment of Diseases, including Muscular Dystrophies |
| title_full_unstemmed | Induced Pluripotent Stem Cells: Hope in the Treatment of Diseases, including Muscular Dystrophies |
| title_short | Induced Pluripotent Stem Cells: Hope in the Treatment of Diseases, including Muscular Dystrophies |
| title_sort | induced pluripotent stem cells hope in the treatment of diseases including muscular dystrophies |
| topic | induced pluripotent stem cells regeneration cellular therapy stem cells muscular dystrophy |
| url | https://www.mdpi.com/1422-0067/21/15/5467 |
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