Genetically modified rat models for severe combined immunodeficiency (SCID)
AssigneeTransposagen Biopharmaceuticals, Inc.
InventorsEric M. Ostertag, John Stuart Crawford, Joseph Ruiz
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Abstract
This invention relates to the engineering of animal cells, preferably mammalian, more preferably rat, that are deficient due to the disruption of tumor suppressor gene(s) or gene product(s). In another aspect, the invention relates to genetically modified rats, as well as the descendants and ancestors of such animals, which are animal models of human cancer and methods of their use.
Claims5
A genetically modified rat or its progeny, whose genome comprises a homozygous disruption of the endogenous adenosine deaminase (Ada) gene and lacks expression of functional Ada protein and wherein said genetically modified rat or its progeny exhibits a severe combined immunodeficiency (SCID) phenotype characterized by no mature populations of a cell population selected from the group consisting of T-, B- and NK cells relative to the rat or its progeny not comprising the genetic modification.
Dependencies are inferred from the claim text.