Yıl 2019, Cilt 20 , Sayı , Sayfalar 17 - 32 2019-02-01

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

Fatima Aerts-Kaya [1] , Gülen Güney Esken [2] , Özgür Doğuş Erol [3]


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.

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.

Gene Therapy, hematopoietic stem cells, lentiviral vectors, biosafety, inherited monogenic diseases
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Birincil Dil en
Konular Biyoloji
Bölüm Derleme/Review
Yazarlar

Orcid: 0000-0002-9583-8572
Yazar: Fatima Aerts-Kaya
Kurum: Hacettepe University Graduate School of Health Sciences, Dept of Stem Cell Sciences
Ülke: Turkey


Orcid: 0000-0002-3158-1587
Yazar: Gülen Güney Esken (Sorumlu Yazar)
Kurum: Hacettepe University Graduate School of Health Sciences, Dept of Stem Cell Sciences
Ülke: Turkey


Orcid: 0000-0001-9301-5401
Yazar: Özgür Doğuş Erol
Kurum: Hacettepe University Graduate School of Health Sciences, Dept of Stem Cell Sciences
Ülke: Turkey


Tarihler

Başvuru Tarihi : 15 Kasım 2018
Kabul Tarihi : 19 Ocak 2019
Yayımlanma Tarihi : 1 Şubat 2019

Bibtex @derleme { trkjnat483402, journal = {Trakya University Journal of Natural Sciences}, issn = {2147-0294}, eissn = {2528-9691}, address = {}, publisher = {Trakya Üniversitesi}, year = {2019}, volume = {20}, pages = {17 - 32}, doi = {10.23902/trkjnat.483402}, title = {HEMATOPOIETIC STEM CELL GENE THERAPY FOR INHERITED MONOGENIC DISEASES AND ITS IMPLICATIONS FOR FUTURE GENE THERAPY TRIALS IN TURKEY}, key = {cite}, author = {Aerts-Kaya, Fatima and Güney Esken, Gülen and Erol, Özgür Doğuş} }
APA Aerts-Kaya, F , Güney Esken, G , Erol, Ö . (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 . DOI: 10.23902/trkjnat.483402
MLA Aerts-Kaya, F , Güney Esken, G , Erol, Ö . "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 (2019 ): 17-32 <https://dergipark.org.tr/tr/pub/trkjnat/issue/39830/483402>
Chicago Aerts-Kaya, F , Güney Esken, G , Erol, Ö . "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 (2019 ): 17-32
RIS TY - JOUR T1 - HEMATOPOIETIC STEM CELL GENE THERAPY FOR INHERITED MONOGENIC DISEASES AND ITS IMPLICATIONS FOR FUTURE GENE THERAPY TRIALS IN TURKEY AU - Fatima Aerts-Kaya , Gülen Güney Esken , Özgür Doğuş Erol Y1 - 2019 PY - 2019 N1 - doi: 10.23902/trkjnat.483402 DO - 10.23902/trkjnat.483402 T2 - Trakya University Journal of Natural Sciences JF - Journal JO - JOR SP - 17 EP - 32 VL - 20 IS - SN - 2147-0294-2528-9691 M3 - doi: 10.23902/trkjnat.483402 UR - https://doi.org/10.23902/trkjnat.483402 Y2 - 2019 ER -
EndNote %0 Trakya University Journal of Natural Sciences HEMATOPOIETIC STEM CELL GENE THERAPY FOR INHERITED MONOGENIC DISEASES AND ITS IMPLICATIONS FOR FUTURE GENE THERAPY TRIALS IN TURKEY %A Fatima Aerts-Kaya , Gülen Güney Esken , Özgür Doğuş Erol %T HEMATOPOIETIC STEM CELL GENE THERAPY FOR INHERITED MONOGENIC DISEASES AND ITS IMPLICATIONS FOR FUTURE GENE THERAPY TRIALS IN TURKEY %D 2019 %J Trakya University Journal of Natural Sciences %P 2147-0294-2528-9691 %V 20 %N %R doi: 10.23902/trkjnat.483402 %U 10.23902/trkjnat.483402
ISNAD Aerts-Kaya, Fatima , Güney Esken, Gülen , Erol, Özgür Doğuş . "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 / (Şubat 2019): 17-32 . https://doi.org/10.23902/trkjnat.483402
AMA Aerts-Kaya F , Güney Esken G , Erol Ö . 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.
Vancouver Aerts-Kaya F , Güney Esken G , Erol Ö . 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. 2019; 20: 32-17.