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Disease models

Additional sex combs like 1
(Asxl1)-mutant mouse model of myelodysplastic syndrome (MDS)

Mice transplanted with bone marrow expressing a mutant form of Asxl1 could model MDS and be used to identify and evaluate new treatments for the condition. Mutations in ASXL1 are found in patients who have MDS, myeloproliferative neoplasms and various types of leukemia. In the model, mice received a bone marrow transplant of cells obtained from mice treated with 5-fluorouracil and transduced with a vector expressing a C-terminal-truncated mutant form of Asxl1. The transplanted mice recapitulated the hallmarks of MDS seen in patients, including multilineage myelodysplasia, pancytopenia and occasional progression to leukemia. Next steps include using the model to evaluate therapies to treat ASXL1-mutant MDS.

SciBX 6(42); doi:10.1038/scibx.2013.1199
Published online Oct. 31, 2013

Unpatented; model available for licensing

Inoue, D. et al. J. Clin. Invest.;
published online Oct. 8, 2013;
doi:10.1172/JCI70739
Contact: Toshio Kitamura, Institute of Medical Science, The University of Tokyo, Tokyo, Japan
e-mail:
kitamura@ims.u-tokyo.ac.jp