Transgenic mouse model for iduronidase deficiency and methods of

Details Transgenic mouse model for iduronidase deficiency and methods of Transgenic mouse model for iduronidase deficiency and methods of

1997-05-20

1999-12-14

Crouch, Deborah

Multicellular living organisms and unmodified parts thereof and

Nonhuman animal

Transgenic nonhuman animal

800 3, 800 9, 800 25, A61K 6700

Patent

active

060020679

ABSTRACT:
A mouse which is homozygous for a disruption in the IDUA gene, but which has normal expression for the SAT-1 gene can be used for evaluating therapeutic agents for use in treating mucopolysaccharidosis Type I, by administering the therapeutic agent to the mouse, and evaluating the mouse for tissue pathology associated with iduronidase deficiency. The mouse can also be used for evaluating the ability of a targeting system to deliver a therapeutic agent to selected tissues or organs by administering an effective iduronidase replacement therapy coupled to the targeting system to a mouse which is homozygous for a disruption in the IDUA gene, but which has normal expression for the SAT-1 gene; and evaluating at least the selected tissue or organ from the mouse for pathology associated with iduronidase deficiency. Targeting systems which can be evaluated using this methodology include targeting moieties which selectively bind to or associate with selected cell types and in vivo and ex vivo gene therapy systems.

REFERENCES:
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Moens, CB, et al. Defects in heart and lung development in compound heterozygotes for two different targeted mutations at the N-myc locus. Development, vol. 119, pp. 485-493, 1993.
Clarke et al., "Mutation Analysis of 19 North American Mucopolysaccharidosis Type I Patients: Identification on Two Additional Frequent Mutations", Human Mutation 3: 275-282 (1994).
Clarke et al., "Murine .alpha.-L-Iduronidase: cDNA Isolation and Expression" Genomics 24: 311-316 (1994).
Clark et al. "Murine Mucopolysaccharidosis Type I: targeted disruption of the murine .alpha.-L-Iduronidase gene", Human Molec Genetics 6: 503-511 (1997).

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