Methods, systems and transgenic animals useful for screening, diagnosing and treating FUS1 related disorders

The FUS1 gene residing in the 3p21.3 chromosome region may function as a tumor suppressor gene. Results show that FUS1 null mutants show consistent changes in NK cells and secreted antibodies, suggesting that FUS1 plays an important role in the development and activation of the mammalian immune system. The invention relates to methods, systems and transgenic animals useful for screening, diagnosing and treating FUS1 related disorders. Interestingly, targeted disruption of FUS1 gene in mice resulted in a viable and fertile phenotype. Possible uses of this invention include using the FUS1 protein to modulate and boost the immune system in diseases like cancer and AIDS. Also, the cDNA and the corresponding protein are small and the applications could include gene therapy with appropriate vectors and protein transduction technology.

U.S. Provisional Application No. 60/697,596 filed 07 Jul 2005 (HHS Reference No. E-137-2005/0-US-01) Inventors: Michael I. Lerman et al. (NCI)

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