Thursday, October 31, 2013
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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.
Published online Oct. 31, 2013
Unpatented; model available
Inoue, D. et al. J.
published online Oct. 8, 2013;
Contact: Toshio Kitamura, Institute of Medical Science, The
University of Tokyo, Tokyo, Japan
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