# US 7,989,675 B2 · Method of identifying compounds using a transgenic pig model of cystic fibrosis

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- Number: US 7,989,675 B2
- Document ID: US7989675B2
- Status: Granted
- Kind: B2
- Application number: 12/074,632
- Filed: 2008-03-05
- Published: 2009-09-17
- Granted: 2011-08-02
- Earliest claimed: 2007-03-28
- Assignees: University of Iowa Research Foundation; The Curators of the University of Missouri
- Inventors: Michael J. Welsh; Christopher S. Rogers; Randall Prather; John Engelhardt; Ziying Yan
- Web page: https://patentgrep.com/patent/US7989675B2

## CPC

- A01K 67/0278: Knock-in vertebrates, e.g. humanised vertebrates
- C07K 14/4712: {Cystic fibrosis}
- C12N 15/8509: {for producing genetically modified animals, e.g. transgenic}
- A01K 2207/15: Humanized animals
- A01K 2217/00: Genetically modified animals
- A01K 2217/052: inducing gain of function
- A01K 2217/056: due to mutation of coding region of the transgene (dominant negative)
- A01K 2217/075: inducing loss of function, i.e. knock out
- A01K 2217/15: Animals comprising multiple alterations of the genome, by transgenesis or homologous recombination, e.g. obtained by cross-breeding
- A01K 2227/108: Swine
- A01K 2267/0306: Animal model for genetic diseases
- A01K: ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- C07K: PEPTIDES (peptides in foodstuffs A23; obtaining protein compositions for foodstuffs, working-up proteins for foodstuffs A23J; preparations for medicinal purposes A61K; peptides containing beta-lactam rings C07D; cyclic dipeptides not having in their molecule any other peptide link than those which form their ring, e.g. piperazine-2,5-diones, C07D; ergot alkaloids of the cyclic peptide type C07D519/02; macromolecular compounds having statistically distributed amino acid units in their molecules, i.e. when the preparation does not provide for a specific; but for a random sequence of the amino acid units, homopolyamides and block copolyamides derived from amino acids C08G69/00; macromolecular products derived from proteins C08H1/00; preparation of glue or gelatine C09H; single cell proteins, enzymes C12N; genetic engineering processes for obtaining peptides C12N15/00; compositions for measuring or testing processes involving enzymes C12Q; investigation or analysis of biological material G01N33/00)
- C12N: MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA (microbiological testing media C12Q1/00)

## Abstract

The present invention provides transgenic, large non-human animal models of diseases and conditions, as well as methods of using such animal models in the identification and characterization of therapies for the diseases and conditions.

## Claims

### Claim 1

A method of identifying a therapeutic agent that can be used in the treatment of cystic fibrosis, the method comprising administering a candidate therapeutic agent to a transgenic pig whose genome has a homozygous disruption in a cystic fibrosis membrane transporter (CFTR) gene, wherein the pig has a symptom of cystic fibrosis, and monitoring the pig for a symptom of cystic fibrosis, wherein detection of improvement in a symptom of cystic fibrosis indicates the identification of a compound that can be used in the treatment of cystic fibrosis.

### Claim 2 (depends on 1)

The method of claim 1, wherein the symptom of cystic fibrosis is monitored in the lung, pancreas, intestine, liver, or kidney of the pig.