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Nature Medicine, ISSN 1078-8956, 06/2018, Volume 24, Issue 6, pp. 739 - 748
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The American Journal of Gastroenterology, ISSN 0002-9270, 04/2008, Volume 103, Issue 4, pp. 914 - 921
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A therapeutic option for monogenic disorders is gene therapy with ex vivo-transduced autologous hematopoietic stem cells (HSCs). Safety or efficacy studies of... 
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