Mouse Model of Alpha-One Antitrypsin (AAT) Deficiency
AssigneeUniversity of Massachusetts
InventorsChristian Mueller, Florie Borel
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Abstract
Transgenic non-human animals, e.g., rodents, e.g., mice comprising genomic mutations that inactive all of the serpin1A genes and thus lack any functional serpinA1 genes. As a result of the genomic mutations, the animals express no hepatic or circulatory AAT protein. Also provided herein are cells and tissues derived from the transgenic mice.
Claims19
A method of evaluating an effect of a test compound on development of Alpha-One Antitrypsin (AAT)-deficiency lung disease, the method comprising:
- providing a transgenic mouse whose genome comprises inactivating mutations in exon 2 of each of Serpina1a, Serpina1b, Serpina1c, Serpina1d, and Serpina1e genes, and which expresses no hepatic or circulatory AAT protein;
- exposing the mouse to the test compound;
- and
- evaluating a parameter of respiratory physiology in the transgenic mouse.
A method of evaluating an effect of a test compound on development of Alpha-One Antitrypsin (AAT)-deficiency lung disease, the method comprising:
- providing a cell, tissue, or organ from a transgenic mouse whose genome comprises inactivating mutations in exon 2 of each of Serpina1a, Serpina1b, Serpina1c, Serpina1d, and Serpina1e genes, and which expresses no hepatic or circulatory AAT protein;
- exposing the cell, tissue, or organ to the test compound;
- and
- evaluating a level of elastase in the cell, tissue, or organ.
The method of claim 19, comprising exposing the cell, tissue, or organ to a potential therapeutic before, during and/or after exposing the cell, tissue, or organ to the test compound.
Dependencies are inferred from the claim text.