. 2023; 40(2): 118-124 | DOI: 10.4274/tjh.galenos.2023.2022.0318 | |||
In Vitro FVIII-Encoding Transgenic Mesenchymal Stem Cells Maintain Successful Coagulation in FVIII-Deficient Plasma Mimicking Hemophilia ACansu Hemşinlioğlu1, Elif Sibel Aslan2, Cihan Tastan3, Didem Çakırsoy4, Raife Dilek Turan5, Utku Seyis6, Muhammer Elek7, Gözde Sır Karakuş6, Ömur Selin Günaydın6, Selen Abanuz8, Derya Dilek Kançağı6, Bulut Yurtsever6, Koray Yalçın9, Murat Kasap10, Ercüment Ovalı61Acıbadem Labcell Cellular Therapy Laboratory, İstanbul, Türkiye; Molecular Biology and Medical Genetics Department, Biruni University, İstanbul, Türkiye2Molecular Biology and Medical Genetics Department, Biruni University, İstanbul, 3Acıbadem Labcell Cellular Therapy Laboratory, İstanbul, Türkiye; Molecular Biology and Genetics Department, Üsküdar University, İstanbul, Türkiye; Transgenic Cell Technologies and Epigenetic Application and Research Center (TRGENMER), Uskudar University, İstanbul, Türkiye 4Acıbadem Labcell Cellular Therapy Laboratory, İstanbul, Türkiye; Medicine Department, Institute of Karolinska, Stockholm, Sweden 5Acıbadem Labcell Cellular Therapy Laboratory, İstanbul, Türkiye; Biotechnology Department, Yeditepe University, İstanbul, Türkiye; Cell and Gene Therapy Center of Excellence, Yeditepe University, İstanbul, Türkiye 6Acıbadem Labcell Cellular Therapy Laboratory, İstanbul, Türkiye 7Acıbadem Labcell Cellular Therapy Laboratory, İstanbul, Türkiye; Biotechnology Department, Yeditepe University, İstanbul, Türkiye 8Acıbadem Labcell Cellular Therapy Laboratory, İstanbul, Türkiye; Medical Biochemistry Department, Acibadem Mehmet Ali Aydinlar University, İstanbul, Türkiye 9Acıbadem Labcell Cellular Therapy Laboratory, İstanbul, Türkiye; Medical Park Goztepe Hospital, Pediatric Bone Marrow Transplantation Unit, İstanbul, Türkiye; Medical Biotechnology Department, Acıbadem Mehmet Ali Aydinlar University, İstanbul, Türkiye 10Department of Medical Biology, Medical School of Kocaeli University, Kocaeli, Türkiye Objective: Hemophilia A is an X-linked recessive bleeding disorder caused by a deficiency of plasma coagulation factor VIII (FVIII), and it accounts for about 80%-85% of all cases of hemophilia. Plasma-derived therapies or recombinant FVIII concentrates are used to prevent and treat the bleeding symptoms along with FVIIImimicking antibodies. Recently, the European Medicines Agency granted conditional marketing approval for the first gene therapy for hemophilia A. The aim of this study was to determine the effectiveness of coagulation in correcting FVIII deficiency with FVIII-secreting transgenic mesenchymal stem cells (MSCs). İn Vitro FVIII İçeren Transgenik Mezenkimal Kök Hücreler Hemofili A’yı Taklit Eden FVIII İçermeyen Plazmada Başarılı Koagülasyon SağlamaktadırCansu Hemşinlioğlu1, Elif Sibel Aslan2, Cihan Tastan3, Didem Çakırsoy4, Raife Dilek Turan5, Utku Seyis6, Muhammer Elek7, Gözde Sır Karakuş6, Ömur Selin Günaydın6, Selen Abanuz8, Derya Dilek Kançağı6, Bulut Yurtsever6, Koray Yalçın9, Murat Kasap10, Ercüment Ovalı61Acıbadem Labcell Cellular Therapy Laboratory, İstanbul, Türkiye; Molecular Biology and Medical Genetics Department, Biruni University, İstanbul, Türkiye2Molecular Biology and Medical Genetics Department, Biruni University, İstanbul, 3Acıbadem Labcell Cellular Therapy Laboratory, İstanbul, Türkiye; Molecular Biology and Genetics Department, Üsküdar University, İstanbul, Türkiye; Transgenic Cell Technologies and Epigenetic Application and Research Center (TRGENMER), Uskudar University, İstanbul, Türkiye 4Acıbadem Labcell Cellular Therapy Laboratory, İstanbul, Türkiye; Medicine Department, Institute of Karolinska, Stockholm, Sweden 5Acıbadem Labcell Cellular Therapy Laboratory, İstanbul, Türkiye; Biotechnology Department, Yeditepe University, İstanbul, Türkiye; Cell and Gene Therapy Center of Excellence, Yeditepe University, İstanbul, Türkiye 6Acıbadem Labcell Cellular Therapy Laboratory, İstanbul, Türkiye 7Acıbadem Labcell Cellular Therapy Laboratory, İstanbul, Türkiye; Biotechnology Department, Yeditepe University, İstanbul, Türkiye 8Acıbadem Labcell Cellular Therapy Laboratory, İstanbul, Türkiye; Medical Biochemistry Department, Acibadem Mehmet Ali Aydinlar University, İstanbul, Türkiye 9Acıbadem Labcell Cellular Therapy Laboratory, İstanbul, Türkiye; Medical Park Goztepe Hospital, Pediatric Bone Marrow Transplantation Unit, İstanbul, Türkiye; Medical Biotechnology Department, Acıbadem Mehmet Ali Aydinlar University, İstanbul, Türkiye 10Department of Medical Biology, Medical School of Kocaeli University, Kocaeli, Türkiye Amaç: Hemofili A, pıhtılaşma faktörü VIII’in (FVIII) eksikliğine bağlı gelişen, hemofili hastalarının yaklaşık %80-85’ini oluşturan, X’e bağlı resesif geçiş gösteren bir kanama bozukluğudur. Kanama semptomlarını önlemek ve tedavi etmek için plazma kaynaklı tedaviler ya da rekombinant FVIII konsantreleri ile FVIII’i taklit eden monoklonal antikorlar kullanılmaktadır. Son zamanlarda EMA, hemofili A’nın ilk gen tedavisi için koşullu pazarlama onayı vermiştir. Bu çalışmada, hemofili A hastalığından sorumlu olan FVIII eksikliğini düzeltebilmek amacıyla FVIII salgılayan transgenik mezenkimal kök hücreler (MKH) ile koagülasyon etkinliğinin değerlendirilmesi amaçlanmaktadır. Cansu Hemşinlioğlu, Elif Sibel Aslan, Cihan Tastan, Didem Çakırsoy, Raife Dilek Turan, Utku Seyis, Muhammer Elek, Gözde Sır Karakuş, Ömur Selin Günaydın, Selen Abanuz, Derya Dilek Kançağı, Bulut Yurtsever, Koray Yalçın, Murat Kasap, Ercüment Ovalı. In Vitro FVIII-Encoding Transgenic Mesenchymal Stem Cells Maintain Successful Coagulation in FVIII-Deficient Plasma Mimicking Hemophilia A. . 2023; 40(2): 118-124 Corresponding Author: Cansu Hemşinlioğlu |
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