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Myeloproliferative disorder

Retinoic acid receptor (RAR)

Mouse studies suggest RAR antagonists could help treat myelofibrosis. Mice with genetic mutations in the two receptor binding regions of nuclear receptor co-repressor 2
(NCOR2; SMRT) exhibited reduced radial bone growth and features of myelofibrosis including increased expression of fibrosis-inducing thrombopoietin (TPO) in bone marrow, which is normally repressed by the SMRT-RAR repressor complex. In irradiated mice transplanted with SMRT-mutant bone marrow that have the myelofibrosis phenotype, a RAR antagonist normalized TPO levels, bone integrity and fibrosis. Next steps could include testing RAR antagonists in additional mouse models of myeloproliferative disease.
Io Therapeutics Inc. has the RAR antagonist IRX4310 in Phase I trials to treat neutropenia.

SciBX 6(47); doi:10.1038/scibx.2013.1353
Published online Dec. 12, 2013

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Hong, S.-H. et al. Proc. Natl. Acad. Sci. USA; published online Nov. 4, 2013;
Contact: Ronald M. Evans, Salk Institute for Biological Studies, La Jolla, Calif.