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Year 2019, Special Issue: Stem Cell, 17 - 32, 01.02.2019
https://doi.org/10.23902/trkjnat.483402

Abstract

Kök
hücre tedavisi rejeneratif tıp ve restoratif tıp alanlarında yeni tedavilerin
geliştirilmesi için büyük avantaj sağlamaktadır. Böylelikle, kök hücre
tedavilerinin kullanımı ve bunların klinik endikasyonları hücrelerin genetik
modifikasyonu ile geliştirilebilmektedir. Türkiye’deki akraba evlilik oranının
fazla olmasından dolayı, yenidoğan genetik kalıtsal hastalıkların insidası
artmaktadır ve bu durum bir sorun teşkil etmektedir. Bu hastalıkların tedavi
edilmesi; 1) kırsal bölgelerde hastalığın sıklıkla geç teşhisi, 2) Özel
merkezlerin uzak olması, 3) Tedavilerin sık hastane kontrolü gerektirmesi, 4)
Tedavi prosedürlerinin hem pahalı hem de invazif olması nedenleriyle zordur.



Bu makalede nadir kalıtsal monogenik hastalıklar için
hematopoetik kök hücre (HKH) gen tedavisinin güncel durumları ve Türkiye’deki
hastalar için alternatif bir tedavi seçeneği olarak kullanımının avantajları
tartışılacaktır. İmmun yetmezlikler, hemoglobinopatiler, birçok enzim
eksikliklerinde retroviral ve lentiviral gen tedavi klinik çalışma sonuçları,
HKH gen tedavisi alanındaki yeni gelişmeler, güvenlik ve etkinliğin artırılması
ve gelecekteki öneriler tartışılacaktır.

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HEMATOPOIETIC STEM CELL GENE THERAPY FOR INHERITED MONOGENIC DISEASES AND ITS IMPLICATIONS FOR FUTURE GENE THERAPY TRIALS IN TURKEY

Year 2019, Special Issue: Stem Cell, 17 - 32, 01.02.2019
https://doi.org/10.23902/trkjnat.483402

Abstract

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.


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There are 113 citations in total.

Details

Primary Language English
Subjects Structural Biology
Journal Section Review/Derleme
Authors

Fatima Aerts-kaya 0000-0002-9583-8572

Gülen Güney Esken This is me 0000-0002-3158-1587

Özgür Doğuş Erol This is me 0000-0001-9301-5401

Publication Date February 1, 2019
Submission Date November 15, 2018
Acceptance Date January 19, 2019
Published in Issue Year 2019 Special Issue: Stem Cell

Cite

APA Aerts-kaya, F., Güney Esken, G., & Erol, Ö. D. (2019). 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, 20, 17-32. https://doi.org/10.23902/trkjnat.483402
AMA Aerts-kaya F, Güney Esken G, Erol ÖD. HEMATOPOIETIC STEM CELL GENE THERAPY FOR INHERITED MONOGENIC DISEASES AND ITS IMPLICATIONS FOR FUTURE GENE THERAPY TRIALS IN TURKEY. Trakya Univ J Nat Sci. February 2019;20:17-32. doi:10.23902/trkjnat.483402
Chicago Aerts-kaya, Fatima, Gülen Güney Esken, and Özgür Doğuş Erol. “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 20, February (February 2019): 17-32. https://doi.org/10.23902/trkjnat.483402.
EndNote Aerts-kaya F, Güney Esken G, Erol ÖD (February 1, 2019) 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 20 17–32.
IEEE F. Aerts-kaya, G. Güney Esken, and Ö. D. Erol, “HEMATOPOIETIC STEM CELL GENE THERAPY FOR INHERITED MONOGENIC DISEASES AND ITS IMPLICATIONS FOR FUTURE GENE THERAPY TRIALS IN TURKEY”, Trakya Univ J Nat Sci, vol. 20, pp. 17–32, 2019, doi: 10.23902/trkjnat.483402.
ISNAD Aerts-kaya, Fatima et al. “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 20 (February 2019), 17-32. https://doi.org/10.23902/trkjnat.483402.
JAMA Aerts-kaya F, Güney Esken G, Erol ÖD. HEMATOPOIETIC STEM CELL GENE THERAPY FOR INHERITED MONOGENIC DISEASES AND ITS IMPLICATIONS FOR FUTURE GENE THERAPY TRIALS IN TURKEY. Trakya Univ J Nat Sci. 2019;20:17–32.
MLA Aerts-kaya, Fatima et al. “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, vol. 20, 2019, pp. 17-32, doi:10.23902/trkjnat.483402.
Vancouver Aerts-kaya F, Güney Esken G, Erol ÖD. HEMATOPOIETIC STEM CELL GENE THERAPY FOR INHERITED MONOGENIC DISEASES AND ITS IMPLICATIONS FOR FUTURE GENE THERAPY TRIALS IN TURKEY. Trakya Univ J Nat Sci. 2019;20:17-32.

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