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Gene and RNA Therapy Center (GRTC)

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Address: Otfried-Müller-Straße 10
72076 Tübingen

Founding Director

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Prof. Dr. Julia Skokowa

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Scientific coordinator

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Dr. Olga Klimenkova

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AG Bakchoul

Portraitfoto

Prof. Dr. med. Tamam Bakchoul

Institute for Clinical and Experimental Transfusion Medicine

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Gene editing of human pluripotent stem cells (iPSCs) to treat inherited platelet disorders

The Institute for Clinical and Experimental Transfusion Medicine (IKET) has long standing expertise in manufacture and application of conventional blood products as well advance therapeutic Medical Products (ATMPS). At our research group protocols have been developed to produce megakaryocytes from non-G-CSF-stimulated blood donors in order to ex vivo human generate platelets. The main goal of ongoing studies is the large scale production of gen-edited platelets, obtained using the CRISP/Cas9 method, to treat patients with inherited platelet disorders. Moreover, several novel in vivo models have been established to preclinical evaluate platelet disorders. Using humanized mouse models the human thrombopoiesis as well as in vivo human platelet survival and function can be analyzed.


GRT projects

Gene Editing: genetically modified iPSCs to treat inherited platelet to better understand inherited platelet disorders and for platelet transfusion


GRT expertise

iPSCs culture for gene editing

Publications

  • Pelzl L, Singh A, Funk J, Witzemann A, Marini I, Zlamal J, Weich K, Abou-Khalel W, Hammer S, Uzun G, Althaus K, Bakchoul T. Antibody-mediated procoagulant platelet formation in COVID-19 is AKT dependent. J Thromb Haemost. 2022 Feb;20(2):387-398. 
  • Zlamal J, Althaus K, Jaffal H, Häberle H, Pelzl L, Singh A, Witzemann A, Weich K, Bitzer M, Malek N, Göpel S, Bösmüller H, Gawaz M, Mirakaj V, Rosenberger P, Bakchoul T. Upregulation of cAMP prevents antibody-mediated thrombus formation in COVID-19. Blood Adv. 2022 Jan 11;6(1):248-258. 
  • Uzun G, Althaus K, Bakchoul T. No Correlation between Anti-PF4 and Anti-SARS-CoV-2 Antibodies after ChAdOx1 nCoV-19 Vaccination. N Engl J Med. 385(14):1334-1336 (2021). 
  • Uzun G, Althaus K, Singh A, Möller P, Ziemann U, Mengel A, Rosenberger P, Guthoff M, Petzold GC, Müller J, Büchsel M, Feil K, Henkes H, Heyne N, Maschke M, Limpach C, Nagel S, Sachs UJ, Fend F, Bakchoul T. The use of IV immunoglobulin in the treatment of vaccine-induced immune thrombotic thrombocytopenia. Blood. 138(11):992-996 (2021). 
  • Althaus K, Möller P, Uzun G, Singh A, Beck A, Bettag M, Bösmüller H, Guthoff M, Dorn F, Petzold GC, Henkes H, Heyne N, Jumaa H, Kreiser K, Limpach C, Luz B, Maschke M, Müller JA, Münch J, Nagel S, Pötzsch B, Müller J, Schlegel C, Viardot A, Bäzner H, Wolf M, Pelzl L, Warm V, Willinek WA, Steiner J, Schneiderhan-Marra N, Vollherbst D, Sachs UJ, Fend F, Bakchoul T. Antibody-mediated procoagulant platelets in SARS-CoV-2-vaccination associated immune thrombotic thrombocytopenia. Haematologica. 106(8):2170-2179 (2021). 

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