# US 8,785,717 B2 · Genetically modified non-human mammals and cells

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- Number: US 8,785,717 B2
- Document ID: US8785717B2
- Status: Granted
- Kind: B2
- Application number: 11/570,087
- Filed: 2005-06-08
- Published: 2009-08-13
- Granted: 2014-07-22
- Earliest claimed: 2004-06-10
- Assignees: University of Leicester
- Applicants: Teizo Fujita; Hans-Wilhelm Schwaeble; Cordula Margaret Stover
- Inventors: Teizo Fujita; Hans-Wilhelm Schwaeble; Cordula Margaret Stover
- Web page: https://patentgrep.com/patent/US8785717B2

## CPC

- A01K 67/0278: Knock-in vertebrates, e.g. humanised vertebrates
- A01K 67/0276: Knock-out vertebrates
- C07K 16/2851: {against the lectin superfamily, e.g. CD23, CD72}
- C07K 16/40: against enzymes
- C12N 9/6424: {Serine endopeptidases (3.4.21)}
- C12N 15/8509: {for producing genetically modified animals, e.g. transgenic}
- A01K 2207/15: Humanized animals
- A01K 2217/00: Genetically modified animals
- A01K 2217/072: maintaining or altering function, i.e. knock in
- A01K 2217/075: inducing loss of function, i.e. knock out
- A01K 2227/105: Murine
- A01K 2267/01: Animal expressing industrially exogenous proteins
- A01K 2267/03: Animal model, e.g. for test or diseases
- A61K 38/00: Medicinal preparations containing peptides (peptides containing beta-lactam rings A61K31/00; cyclic dipeptides not having in their molecule any other peptide link than those which form their ring, e.g. piperazine-2,5-diones, A61K31/00; ergot alkaloids of the cyclic peptide type A61K31/48; containing macromolecular compounds having statistically distributed amino acid units A61K31/74; medicinal preparations containing antigens or antibodies A61K39/00; medicinal preparations characterised by the non-active ingredients, e.g. peptides as drug carriers, A61K47/00)
- C07K 2317/14: Specific host cells or culture conditions, e.g. components, pH or temperature
- C12N 2510/00: Genetically modified cells
- C12N 2517/00: Cells related to new breeds of animals
- C12N 2800/30: Vector systems comprising sequences for excision in presence of a recombinase, e.g. loxP or FRT
- 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)
- A61K: PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES (devices or methods specially adapted for bringing pharmaceutical products into particular physical or administering forms A61J3/00; chemical aspects of, or use of materials for deodorisation of air, for disinfection or sterilisation, or for bandages, dressings, absorbent pads or surgical articles A61L; soap compositions C11D)

## Abstract

Genetically modified mammals are described which lack the mannan binding lectin associated serine protease MASP-2, together with methods and constructs for their production. Such mammals are useful as models for disorders of the complement system, and in the identification of treatments for such disorders. Also described are mammals which lack the associated protein MAp19; such mammals may also lack MASP-2.

## Claims

### Claim 1

A transgenic rodent or transgenic rodent cell, wherein the gene encoding endogenous MASP-2 polypeptide and/or the gene encoding endogenous MAp19 polypeptide is/are absent or partially absent or disrupted in said rodent or said rodent cell, so that endogenous MASP-2 polypeptide and/or endogenous MAp19 polypeptide is not produced, wherein the transgenic rodent lacks a lectin complement pathway response and retains a classical complement pathway response.

### Claim 2 (depends on 1)

The transgenic rodent cell according to claim 1, wherein all endogenous MASP-2 and/or endogenous MAp19 gene sequences are absent from the genome.

### Claim 3 (depends on 1)

The transgenic rodent cell according to claim 1, wherein the cell is obtained by targeted deletion of essentially all endogenous MASP-2 and/or MAp 19 gene sequences.

### Claim 4 (depends on 1)

The transgenic rodent or rodent cell of claim 1, wherein the rodent cell is a mouse or mouse cell.

### Claim 5 (depends on 1)

The transgenic rodent cell according to claim 1, wherein the cell is an embryonic stem cell.

### Claim 6 (depends on 1)

The transgenic rodent or rodent cell of claim 1, wherein exons 10, 11, and 12 of the endogenous MASP-2 gene and/or the exon 5 of the endogenous MAp19 gene are deleted from the genome.

### Claim 7 (depends on 1)

The transgenic rodent or rodent cell of claim 1, wherein one or more selectable marker(s) selected from a neomycin resistance gene, a puromycin resistance gene, and/or a hygromycin resistance gene are present within the genome.