Transgenic animals lacking prion proteins
OwnerCharles Weissmann, Hansruedi Bueler +3inventors
InventorsCharles Weissmann, Hansruedi Bueler, Michel Aguet, Marek Fischer, Andreas Sailer
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Abstract
This invention relates to transgenic mammals and birds that are not susceptible to spongiform encephalopathies (i.e., scrapie-like or prion diseases), due to absence of endogenous functional prion protein ("PrP"). More particularly, this invention relates to DNA targeting molecules that specifically disrupt PrP genes by homologous recombination in transfected animal cells, to cultured cells transformed with such DNA targeting molecules, and to animals derived from those transformed cells and the transgenic progeny of such animals.
Claims7
A transgenic mouse homozygous for endogenous prion gene disruption, said disruption resulting from insertion of a selectable marker gene sequence or other heterologous sequences into the genome by homologous recombination,
- wherein said disruption prevents expression of functional endogenous prion protein, and,
- wherein lack of expression of endogenous prion protein renders said mouse unsusceptible to prion diseases and replication of the infectious agent.
A DNA targeting molecule capable of specifically functionally disrupting expression of an endogenous gene encoding prion protein in mammalian cells wherein disruption of said prion-encoding gene occurs by homologous recombination.
A method of disrupting expression of functional endogenous prion protein in a mammalian cell comprising the step of disrupting one or more endogenous genes encoding prion proteins by homologous recombination with a DNA targeting molecule specific for the gene and capable of specifically functionally disrupting expression of said endogenous gene.
Dependencies are inferred from the claim text.