Description of Invention: Myelodysplastic syndrome (MDS) is collection of closely related blood diseases that arise in the bone marrow characterized by anemia, neutropenia, and thrombocytopenia resulting from hematopoietic stem cell disorders. A variety of genetic aberrations have been associated with MDS, including chromosomal translocations of the NUP98 gene. The only current curative therapy for MDS is allogeneic bone marrow transplant. Without bone marrow transplant, patients either die of progressive pancytopenia or following transformation of MDS to acute myeloid leukemia. Progress in understanding and treating MDS has been hampered by a lack of an animal model that accurately recapitulates all of the features of human MDS. Utilizing a NUP98-HOXD13 (hereafter NHD13) fusion gene, a mouse model was developed to elucidate the biology of MDS. Genetically engineered mice that express an NHD13 transgene display all of the phenotypic features of MDS including peripheral blood cytopenia, bone marrow dysplasia, and transformation to acute leukemia. These mice provide an accurate preclinical model for MDS.
Applications: Model to study MDS and evaluate MDS therapy
Development Status: The technology is currently in the pre-clinical stage of development.
Patent Status: Research Tool -- patent protection is not being pursued for this technology
YW Lin et al. Notch1 mutations are important for leukemic transformation in murine models of precursor-T leukemia/lymphoma. Blood. 2006 Mar 15;107(6):2540-2543. [ PubMed abs ]
YW Lin et al., NUP98-HOXD13 transgenic mice develop a highly penetrant, severe myelodysplastic syndrome that progresses to acute leukemia. Blood. 2005 Jul 1;106(1):287-295. [ PubMed abs ]
Licensing Status: Available for non-exclusive licensing.
Collaborative Research Opportunity: The Leukemia Biology Section, Genetics Branch, National Cancer Institute is seeking statements of capability or interest from parties interested in collaborative research to further develop, evaluate, or commercialize the NHD13 mouse model. Please contact John D. Hewes, Ph.D. at 301-435-3121 or firstname.lastname@example.org for more information.
Portfolios: Devices/Instrumentation Devices/Instrumentation - Research Tools and Materials Cancer Cancer - Research Materials Internal Medicine Internal Medicine - Research Materials Miscellaneous Animal Model
For Additional Information Please Contact: Jennifer Wong NIH Office of Technology Transfer 6011 Executive Blvd. Suite 325, Rockville, MD 20852 United States Email: email@example.com Phone: 301-435-4633 Fax: 301-402-0220