Journal of Leukemia
Open Access
ISSN: 2329-6917
ISSN: 2329-6917
Myles C Cabot
Tanzania
Research Article
Engraftment of Human Primary Acute Myeloid Leukemia Defined by Integrated Genetic Profiling in NOD/SCID/IL2rγnull Mice for Preclinical Ceramide-Based Therapeutic Evaluation
Author(s): Brian M Barth, Nichole R Keasey, Xujung Wang, Sriram S Shanmugavelandy, Raajit Rampal, Todd Hricik, Myles C Cabot, Mark Kester, Hong- Gang Wang, Leonard D Shultz, Martin S Tallman, Ross L Levine, Thomas P Loughran Jr and David F ClaxtonBrian M Barth, Nichole R Keasey, Xujung Wang, Sriram S Shanmugavelandy, Raajit Rampal, Todd Hricik, Myles C Cabot, Mark Kester, Hong- Gang Wang, Leonard D Shultz, Martin S Tallman, Ross L Levine, Thomas P Loughran Jr and David F Claxton
Acute Myeloid Leukemia (AML) is a highly heterogeneous and poor prognosis disease with few available therapeutic options. Novel advances are urgently needed, however effective models to test experimental therapeutics have been lacking. Recently, NOD/SCID/IL2r?null (NSG) mice were shown to engraft primary human AML in a manner that recapitulated the natural disease and its progression. Additionally, integrated genomic profiling was used to refine risk stratification of AML. In this study, we demonstrated the engraftment of molecularly defined primary AML in NSG mice. We showed that AML that express DNMT3A mutations, which predict for adverse outcome, engrafted with exceptional efficacy. Lastly, we demonstrated that human AML-engrafted NSG mice can be effectively used to study novel ceramide-based therapeutics. Ceramide is a bioactive sphingolipid that has been implicated as an inducer of .. View More»
DOI:
10.4172/2329-6917.1000146