HEMATOPOIETIC STEM CELL GENE THERAPY FOR INHERITED MONOGENIC DISEASES AND ITS IMPLICATIONS FOR FUTURE GENE THERAPY TRIALS IN TURKEY
Stem cell therapy offers a great advantage for the development of new treatments in the field of regenerative and restorative medicine. However, the use of stem cell therapies and their clinical indications can even be further improved using genetic modification of the cells. Due to the high level o...
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| Format: | Article |
| Language: | English |
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Trakya University
2019-02-01
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| Series: | Trakya University Journal of Natural Sciences |
| Subjects: | |
| Online Access: | https://dergipark.org.tr/en/download/article-file/636889 |
| _version_ | 1827767495443873792 |
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| author | Fatima Aerts-kaya Özgür Doğuş Erol Gülen Güney Esken |
| author_facet | Fatima Aerts-kaya Özgür Doğuş Erol Gülen Güney Esken |
| author_sort | Fatima Aerts-kaya |
| collection | DOAJ |
| description | Stem cell therapy
offers a great advantage for the development of new treatments in the field of
regenerative and restorative medicine. However, the use of stem cell therapies
and their clinical indications can even be further improved using genetic modification
of the cells. Due to the high level of consanguineous marriages in Turkey, the
country suffers from an increased frequency of inborn genetically inherited diseases.
Treatment of these diseases is difficult, since 1) diagnosis is often delayed
in rural areas, 2) distance to specialized centers may be considerable, 3)
treatment may require frequent hospital visits and 4) treatment procedures are
often both invasive and expensive. Here, we discuss the current status of gene therapy of hematopoietic stem cells
(HSCs) for rare, inherited monogenic diseases and the advantages to use these
cells as an alternative treatment option for patients in Turkey. We discuss
results of clinical trials using retroviral and lentiviral gene therapy for the
treatment of immune deficiencies, hemoglobinopathies and several enzyme
deficiencies, new developments in the field of the HSC gene therapy to improve
safety and efficacy and recommendations for the future. |
| first_indexed | 2024-03-11T12:00:23Z |
| format | Article |
| id | doaj.art-0ec320dc592d48feb65de1b7cd4b03fb |
| institution | Directory Open Access Journal |
| issn | 2528-9691 |
| language | English |
| last_indexed | 2024-03-11T12:00:23Z |
| publishDate | 2019-02-01 |
| publisher | Trakya University |
| record_format | Article |
| series | Trakya University Journal of Natural Sciences |
| spelling | doaj.art-0ec320dc592d48feb65de1b7cd4b03fb2023-11-08T07:27:56ZengTrakya UniversityTrakya University Journal of Natural Sciences2528-96912019-02-0120173210.23902/trkjnat.48340269HEMATOPOIETIC STEM CELL GENE THERAPY FOR INHERITED MONOGENIC DISEASES AND ITS IMPLICATIONS FOR FUTURE GENE THERAPY TRIALS IN TURKEYFatima Aerts-kaya0Özgür Doğuş Erol1Gülen Güney Esken2Hacettepe University Graduate School of Health Sciences, Dept of Stem Cell SciencesHacettepe University Graduate School of Health Sciences, Dept of Stem Cell SciencesHacettepe University Graduate School of Health Sciences, Dept of Stem Cell SciencesStem cell therapy offers a great advantage for the development of new treatments in the field of regenerative and restorative medicine. However, the use of stem cell therapies and their clinical indications can even be further improved using genetic modification of the cells. Due to the high level of consanguineous marriages in Turkey, the country suffers from an increased frequency of inborn genetically inherited diseases. Treatment of these diseases is difficult, since 1) diagnosis is often delayed in rural areas, 2) distance to specialized centers may be considerable, 3) treatment may require frequent hospital visits and 4) treatment procedures are often both invasive and expensive. Here, we discuss the current status of gene therapy of hematopoietic stem cells (HSCs) for rare, inherited monogenic diseases and the advantages to use these cells as an alternative treatment option for patients in Turkey. We discuss results of clinical trials using retroviral and lentiviral gene therapy for the treatment of immune deficiencies, hemoglobinopathies and several enzyme deficiencies, new developments in the field of the HSC gene therapy to improve safety and efficacy and recommendations for the future.https://dergipark.org.tr/en/download/article-file/636889gene therapyhematopoietic stem cellslentiviral vectorsbiosafetyinherited monogenic diseases |
| spellingShingle | Fatima Aerts-kaya Özgür Doğuş Erol Gülen Güney Esken HEMATOPOIETIC STEM CELL GENE THERAPY FOR INHERITED MONOGENIC DISEASES AND ITS IMPLICATIONS FOR FUTURE GENE THERAPY TRIALS IN TURKEY Trakya University Journal of Natural Sciences gene therapy hematopoietic stem cells lentiviral vectors biosafety inherited monogenic diseases |
| title | HEMATOPOIETIC STEM CELL GENE THERAPY FOR INHERITED MONOGENIC DISEASES AND ITS IMPLICATIONS FOR FUTURE GENE THERAPY TRIALS IN TURKEY |
| title_full | HEMATOPOIETIC STEM CELL GENE THERAPY FOR INHERITED MONOGENIC DISEASES AND ITS IMPLICATIONS FOR FUTURE GENE THERAPY TRIALS IN TURKEY |
| title_fullStr | HEMATOPOIETIC STEM CELL GENE THERAPY FOR INHERITED MONOGENIC DISEASES AND ITS IMPLICATIONS FOR FUTURE GENE THERAPY TRIALS IN TURKEY |
| title_full_unstemmed | HEMATOPOIETIC STEM CELL GENE THERAPY FOR INHERITED MONOGENIC DISEASES AND ITS IMPLICATIONS FOR FUTURE GENE THERAPY TRIALS IN TURKEY |
| title_short | HEMATOPOIETIC STEM CELL GENE THERAPY FOR INHERITED MONOGENIC DISEASES AND ITS IMPLICATIONS FOR FUTURE GENE THERAPY TRIALS IN TURKEY |
| title_sort | hematopoietic stem cell gene therapy for inherited monogenic diseases and its implications for future gene therapy trials in turkey |
| topic | gene therapy hematopoietic stem cells lentiviral vectors biosafety inherited monogenic diseases |
| url | https://dergipark.org.tr/en/download/article-file/636889 |
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