mayo clinic ventures 2013 - microsoft · 2015. 7. 25. · 1- breast cancer, triple neatie, brain...

Antibodies . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . p .1HumAn Cell lines . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . p .6HumAn tumor xenogrAfts . . . . . . . . . . . . . . . . . . . . . . p .7pHArmACogenomiC sCreening pAnel . . . . . . . . . . . . . . p .7trAnsgeniC miCe . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . p .8

mAyo CliniC ventures

rese ArCH re Ag ents AvA ilAb le for l iCens ing

2013

AntibodiesmAyo CAse # Antibody nAme Clone type epitope known

AppliCAtionsknown reACtivity

pubmed id #

2006-213 AGR2 Anterior gradient

2

-- Rabbit polyclonal Amino acids

21-175

IHC, WB Human 15834940

2001-030 β Amyloid β Amyloid mIg2.1 Mouse

monoclonal

Aβ 1-42 -- Human --

2006-139 β Amyloid β Amyloid IgG4.1, PC4.1 Mouse

monoclonal

Aβ1-42 IHC Human 17596213

19775170

2008-103 β Amyloid β Amyloid MM43-14.1.1 Mouse

monoclonal

Aβ 1-38 ELISA, IHC, IP,

WB

Human --


monoclonal


WB

Rat --

2008-120 β Amyloid β Amyloid MM32-13.1.1

Ab40

Mouse

monoclonal

IgG1k


WB

Human 17234594


monoclonal

IgG1k


WB

Human --


monoclonal

IgG1k

Aβ 1-42 ELISA, IHC Human --

2008-123 β Amyloid β Amyloid Ab-9 Mouse

monoclonal

IgG2ak


WB

Human --

2008-124 β Amyloid β Amyloid AB42-5 Mouse

monoclonal

IgG2bk


WB

Human --

2008-125 β Amyloid β Amyloid MM26-2.1.3

Ab42.2

Mouse

monoclonal

IgG1k


WB

Human --


monoclonal

IgG2ak


2008-139 β Amyloid β Amyloid Ab9 15E7.17.10 Mouse

monoclonal IgA


2008-346 β Amyloid β Amyloid AB42-2 Mouse

monoclonal IgG3


1987-046 BrdU Bromo-

deoxyuridine

BU-1 Mouse

monoclonal

IgG2a

-- FC, IHC Human 3905299

elisA - enzyme-linked immunosorbent assay, fC - flow cytometry, iHC - immunohistochemistry, ip - immunoprecipitation, riA - radioimmunoassay, wb - western blot 1

2012-253 C9ORF72 C9ORF72 -- Rabbit polyclonal Poly-glycine-

proline residues


2005-031 Caspase 8 p41 p41 fragment of

caspase 8

-- Mouse

monoclonal

C-terminus IHC, IP, WB Human 17442709

2008-114 Caveolin-1 Caveolin-1 -- Rabbit polyclonal -- IHC, IP, WB Human 19641024

2010-209 CCDC53 Coiled-coil

domain-

containing

protein 53


1-194

WB Human 20498093

2006-059 CIN85 Cbl-interacting

protein of 85

kDa


291-400

IHC, IP, WB Human 20711168

2010-113 CIP4 cdc42-

interacting

protein 4


297-321

IHC, IP, WB Human --

2010-113 COTL1 Coactosin-like

protein

-- Rabbit polyclonal -- IHC, IP, WB Human --

2011-076 Ect2 (P)T328 Phosphorylated

epithelial cell

transforming

sequence 2

-- Rabbit polyclonal Ect2 peptides

phosphorylated

at T328

WB Human 21189248

2008-115 Eps15 EGFR pathway

substrate clone

15


485-510 and

855–882


2009-333 ERβ Estrogen

receptor beta

-- Mouse

monoclonal

Amino acids

1-140

ELISA, IHC, WB Human --

2006-059 Evl Ena/VASP-like

protein

-- Rabbit polyclonal -- IHC, IP, WB Human --

2010-226 FAM21 (A,B,C) WASH complex

subunits FAM

21A-C

-- Rabbit polyclonal FAM21C amino

acids 811-1002

IHC, WB Human 19922874,

20498093

2010-113 FHOD1 Formin-

homology-

2-domain

containing

protein 1


335-360 and

1072-1095


2006-219 FKBP51 FK506-binding

immunophilin,

51kDa

Hi51B Mouse

monoclonal

-- IP, WB Human, Mouse,

Rabbit

--

mAyo CAse # Antibody nAme Clone type epitope known AppliCAtions

known reACtivity

pubmed id #

Antibodies Continued

2 elisA - enzyme-linked immunosorbent assay, fC - flow cytometry, iHC - immunohistochemistry, ip - immunoprecipitation, riA - radioimmunoassay, wb - western blot


known reACtivity

pubmed id #

2006-220 FKBP52 FK506-binding

immunophilin,

52kDa

Hi52C Mouse

monoclonal

-- IP, WB Human, Mouse,

Rabbit

15831525

2008-233 Frataxin Frataxin PAC2517,

PAC2518

Rabbit polyclonal Amino acids

56-210

WB, IP Human 20889968

2007-323 GCUNC45 General cell

UNC45

AbS1 Mouse

monoclonal

-- IHC, WB Human 16478993

2010-113 GEF-H1 Guanine

nucleotide

exchange factor

H1


718-742 and

678-789


2006-059 HS1 Hematopoietic

cell specific Lyn

substrate 1


330-407


2006-107 HtrA1 HtrA serine

peptidase 1


161-480

ELISA Human 16767218

2012-093 LRP-1 Low-density

lipoprotein

receptor-related

protein 1

6F8 Mouse

monoclonal

IgG1k

C-terminus

IHC,WB Human, Mouse --

1999-064 MBP-1 Major basic

protein

-- Rabbit polyclonal -- IHC, IF, WB Human, Mouse --


protein

14.7.4 Rat monoclonal -- IHC, WB Mouse 11067904


protein 2

J191-12H11 Mouse

monoclonal

Amino acids

116-131

RIA, WB Human 10318872

2009-132 MDC1 Mediator of

DNA damage

checkpoint 1

P2B11 Mouse

monoclonal

IgG1k

N-terminus IHC, IP, WB Human 12607004

2001-021 NIS Sodium-iodide

transporter

2-2 Mouse

monoclonal

Amino acids

37-54



transporter

14f Mouse

monoclonal

Amino acids

625–643



transporter

FP-5A Mouse

monoclonal

IgG1k

Amino acids

468-643


2002-076 nPS1 Presenilin 1 -- Rabbit polyclonal N-terminus WB Mouse 8878479



known reACtivity

pubmed id #

2006-231 Orai1 CRAC channel

subunit

MC1849 Rabbit polyclonal Amino acids

201-218

(extracellular)


2006-231 Orai1 CRAC channel

subunit

MC1850 Rabbit polyclonal Amino acids 22-

40 (intracellular)


1997-094 PHAS-1/4E-BP1 PHAS-1/4E-BP1 -- Rabbit polyclonal -- WB Human --

1996-109 ProMBP1 Pro-major basic

protein 1

J174-7D4 Mouse

monoclonal

IgG1k

Amino acids

78-105


2006-072

2006-272

Pyk2 Proline-rich

tyrosine kinase 2

12A10 Mouse

monoclonal

FERM F3

subdomain

WB, ELISA Human --

2005-211 Secretin

receptor splice

variant

Secretin

receptor splice

variant

8F4 Mouse

monoclonal

Amino acids

61-74 of splice

variant

ELISA, WB Human 17678920

2005-211 Secretin

receptor splice

variant

Secretin

receptor splice

variant

5G1 Mouse

monoclonal

Amino acids

82-97 of splice

variant


2004-287 Smad3 (P) Phosphorylated

Smad3

-- Rabbit polyclonal COOH-

GSPSIRCSpSVpS

WB Human 15520863

2011-158 SNAI1 (P)S11 Phosphorylated

SNAI1

-- Rabbit polyclonal -- IHC, WB Human 22276203

2006-059 STIM1 Stromal

interaction

molecule


657-685


2006-231 STIM1 CRAC channel

subunit

MC1863 Rabbit polyclonal Amino acids

69-89


2010-209 Strumpellin Strumpellin -- Rabbit polyclonal Amino acids

696-820

WB Human 20498093

2010-209 SWIP WASH complex

subunit 7


1-93 and 928-

1173

WB Human 20498093

2007-103 TDP-43 TAR DNA-binding

protein-43

-- Rabbit polyclonal C-terminus ELISA, IHC, IP,

WB

Human, Mouse --

1995-072 TIEF-1/KLF10 TGF-β Inducible

Early Factor-1

-- Rabbit polyclonal Multiple

antibodies and

epitopes

IHC, WB Human --

Antibodies Continued



known reACtivity

pubmed id #

2010-113 TOCA-1 Transducer

of cdc42-

dependent actin

assembly


296-319


2011-261 TRAIL-short TNF-related

apoptosis

inducing ligand

(short)

2E5 Mouse

monoclonal

C-terminal 11

amino acids


2007-324 tsp23 Transcript like

p23

JJ6 Mouse

monoclonal

-- WB Human 8114727

2006-059 TUBA Tubulin alpha -- Rabbit polyclonal Amino acids

365-384


2006-059 VASP Vasodilator-

stimulated

phosphoprotein


231-325


2002-136 Vav-3 Vav-3 -- Rabbit polyclonal -- IP, WB Human, Mouse 12234921

1995-095 Vitamin D

receptor

Vitamin D

receptor

24.8, 24.45,

42.11, 46.40,

68.30, 80.6 and

80.12

Mouse

monoclonal

Multiple

antibodies and

epitopes

WB Human 7692846

2010-113 Vps35 Anti-Vesicle

protein sorting

35


461-796

IHC, WB Human --

2010-113 WASH WAS protein

family homolog

1


1-168

WB Human 19922874

2010-113 WASH WAS protein

family homolog

1


316-468


2006-059 WAVE2 Wiskott-Aldrich

syndrome

protein family

member 2


185-265 and

350-498



human cell linesmayo case # name Tissue source DescripTion

2007-050 AL amyloidosis immunoglobulin

lambda secreting cell lines

Bone marrow ALMC-1 and ALMC-2 cell lines were isolated from an AL amyloidosis patient before and after

receiving a stem cell transplant, respectively. Secreted light chains contain beta structure

necessary for amyloid aggregation.

2010-305 Breast cancer, triple negative,

brain metastasis

Metastasis Short tandem DNA repeat analysis was performed and confirms parental tumor match.

Mouse tumor graft available. Multiple cell lines are available.

2010-203 Bronchopulmonary carcinoid

tumor lines

Lung Three cell lines have been well characterized, form tumors in nude mice, and serve as

excellent models for study of this rare tumor type.

2007-259 CVID patient B cell lines with

TACI mutation

Peripheral blood EBV transformed B cells from two patients with common variable immunodeficiency (CVID)

that carry the TACI A181E mutation. Useful for biochemical and functional characterization of

the mutation.

1993-010 Fetal osteoblast progenitor

(hFOB) cell lines

Fetal limb tissue

immortalized with mutant

SV40 large T antigen

Mutated large T antigen conveys temperature sensitive phenotype. Cells grow rapidly at low

temperature, differentiate and mineralize bone marrow at high temperature. Useful for the study

of osteoblasts, osteoporosis, transplants, and cancer.

2007-235 Ileal and rectal carcinoid tumor

cell lines

Liver metastases These cell lines are carcinoid tumor cells transformed with SV40 large T antigen. They are

useful as models for the study of intestinal carcinoid tumors.

1988-049 Mast cell leukemia line

(HMC-1)

Peripheral blood Unique cell line useful for the study of mast cell biology. Derived from a patient with mast

cell leukemia.

2010-305 Melanoma Lymph node metastasis

1997-106 Multiple myeloma cell lines ANBL-

6, DP-6, KAS-6/1, and KP-6

Bone marrow, pleural

effusion, peripheral blood

The cell lines have maintained IL-6 responsiveness and are useful for studying multiple

myeloma in culture and in vivo models.

2011-113 Multiple myeloma cell line

JMW

Peripheral blood This IL-6 dependent cell line expresses IgA lambda and contains a t(4;14) translocation.

Suitable for the study of multiple myeloma cell biology.

2010-305 Ovarian serous carcinoma

and normal primary ovarian

epithelial cells

Ovary Short tandem DNA repeat analysis was performed and confirms parental tumor match. This

line is also paclitaxel resistant and available expressing luciferase.

2010-305 Renal cell carcinoma (clear

cell) and normal primary renal

epithelial cells

Kidney and adrenal

metastasis

Stage II, stage IV primary tumor, and right adrenal metastasis of the stage IV tumor. Short

tandem DNA repeat analysis was performed and confirms parental tumor match in stage IV

primary tumor line.

2012-133 Sinusoidal endothelial cell line Liver EBV transformed hepatic sinusoidal endothelial cells maintain an endothelial phenotype

including formation of lamellipodia and filopodia and a cobblestone morphology of cell

monolayers. Useful for studying liver endothelial cell functions.

2008-277 Thyroid carcinoma (anaplastic)

cell lines

Thyroid Multiple cell lines representing all histological subtypes of anaplastic thyroid carcinoma. Cell

lines have been extensively characterized for several oncogenic mutations and thyroid-

specific markers. Useful for evaluating anti-tumor drugs and studying thyroid cancer.

2010-306 Thyroid carcinoma (follicular) Thyroid and lymph node

metastases

Multiple cell lines. Primary tumors and matching metastases available.

2010-306 Thyroid carcinoma (papillary) Multiple cell lines are available.

2010-305 Uterine sarcoma Uterus

2012-121 Waldenstrom

Macroglobulinemia cell line

MWCL-1

Bone marrow Genetic analysis confirms clonal relationship with founding tumor. Cell line secretes high

levels of IgM. Genetic, immunophenotype, and biologic data confirm validity of the cell lines

as a model of WM.


pHArmACogenomiC sCreening pAnelmAyo CAse # nAme desCription utility

2007-064 Human cell line panel for

preclinical pharmacogenomics

testing

Panel is comprised of immortalized lymphoblastoid

cell lines from nearly 300 healthy individuals of

varying ethnicities. For each line, genome-wide SNP

characterization, gene expression, genome wide

copy number, and substantial genomic sequencing

data are available.

This panel is valuable for pharmacogenomic evaluation

of drug efficacy and safety in addition to identification

of drug-specific biomarkers for optimizing therapy and

drug discovery. Panel has been used to identify markers

predicting response to radiation therapy and anti-neoplastic

cytidine analogs.

HumAn tumor xenogrAftsmAyo CAse # tumor type tissue sourCe desCription

2010-206 Bladder cancer Bladder Bladder cancer tumor graft.*

2010-206 Breast cancer Breast tissue and lung, brain, and liver metastases Multiple triple negative (ER-, PR-, Her2/neu-) tumor grafts

available.*

2010-206 Clear cell renal cell carcinoma Kidney, adrenal gland metastasis Stage I, III, and IV tumors with some matching primary

tumors and metastases available.*

2003-128 Glioblastoma Brain Numerous, well-characterized tissue grafts are available.

2010-206 Lung cancer Lung Tumor grafts available from several tumor subtypes.

2010-206 Melanoma Skin, lymph node metastasis Multiple tumor grafts available.*

2012-107 Ovarian Ovary Tumor grafts available from several tumor subtypes.**

2010-206 Pancreatic cancer Liver metastases Liver metastases of acinar cell pancreatic cancer available.*

2013-061 Pancreatic cancer Pancreas A large number of characterized grafts are available.

2010-206 Thyroid cancer Thyroid Anaplastic thyroid carcinoma, thyroid squamous carcinoma,

and papillary thyroid carcinoma tissues available.*

* Freshly resected human tumor tissue was implanted subcutaneously into the right flank of nude mice.

**Freshly resected human tissue was injected intraperitoneally into mice.


transgenic mice (allergic disease models)mayo case # description genotype/phenotype Utility pUblications

1995-138 Mouse model for eosinophil-

mediated cutaneous disease

IL-5 overexpression is driven

by keratinocyte (skin) specific

regulatory sequences. These

mice (Ker14/IL-5) have increased

numbers of eosinophils in the skin

and blood.

Model of allergic skin disorders

including atopic dermatitis and

skin disorders characterized by

hypereosinophilia.

1995-139 Mouse model for

hypereosinophilic syndromes

These mice (NJ1638) express IL-5

in peripheral T cells.

Study of hypereosinophilic

syndromes.

Lee NA, et al. Expression of IL-5

in thymocytes/T cells leads to the

development of a massive eosinophilia,

extramedullary eosinophilopoiesis, and

unique histopathologies. J Immunol

1997;158(3):1332-44.

1995-140 Mouse model for asthma

and eosinophilic pulmonary

inflammation

Generated using the CC10

promoter to express IL-5. These

mice (CCIL5) show hyperreactivity

to methacholine challenge and

eosinophil infiltration into lung

epithelium.

Model of eosinophilic

pulmonary inflammation.

Lee J, et al. Interleukin-5 expression in the

lung epithelium of transgenic mice leads to

pulmonary changes pathognomic of asthma J

Exp Med 1997;185(12):2143-56.

Borchers MT, et al. Intrinsic AHR in IL-5

transgenic mice is dependent on CD4(+) cells

and CD49d-mediated signaling. Am J Physiol

Lung Cell Mol Physiol 2001; 281(3): L653-9.

1998-039 Major basic protein (MBP)

knockout mouse for the

study of asthma and related

conditions

Mouse strain which does not

express the eosinophil granule

major basic protein (MBP).

Useful for the study of asthma

and asthma-related conditions,

including therapeutic screening.

Denzler K, et al. Eosinophil major basic

protein-1 does not contribute to allergen-

induced airway pathologies in mouse models

of asthma. J Immunol 2000;165(10):5509-17.

2003-196 Mouse line (PHIL) devoid of

eosinophils for the study of

eosinophils and their roles in

diseases

Eosinophil-specific regulatory

sequences were used in

conjunction with the diphtheria

toxin A gene to eliminate

production of eosinophils.

Model for examining the role of

eosinophils in various diseases.

Lee JJ, et al. Defining a link with asthma in

mice congenitally deficient in eosinophils.

Science 2004;305(5691):1773-6.

Jacobsen EA, et al. Allergic pulmonary

inflammation in mice is dependent on

eosinophil-induced recruitment of effector T

cells. J Exp Med 2008;205(3):699-710.

2010-196 iPhil mouse line permitting

inducible loss of eosinophils

to study eosinophil-related

diseases

Inducible ablation of eosinophils is

accomplished through expression

of the human diptheria toxin

receptor exclusively in cells of the

eosinophil lineage. Diptheria toxin

exposure completely eliminates

circulating eosinophils without

affecting other cell types. Depletion

is reversible in days.

Model for examining the role

of eosinophil recruitment

and activation in allergy,

gastrointestinal disease,

cardiovascular disease, and

transplant rejection.


transgenic mice (autoimmune disease models)mayo case # description genotype/phenotype utility publications

1997-069 HLA-DR2 transgenic mouse

for the study of autoimmune

disease

These mice express HLA-DR2

in a mouse class II-deficient

background.

Model system for the study of

autoimmune disease.

Gonzalez-Gay MA, et al. Human leukocyte

antigen-DRB1*1501 (DR2Dw12) transgene

reduces incidence and severity of arthritis in

mice. Hum Immunol 1996;50(1):54-60.

Mangalam AK, et al. HLA class II transgenic

mice mimic human inflammatory diseases.

Adv Immunol 2008;97:65-147.

Paisansinsup T, et al. HLA-DR modulates

autoantibody repertoire, but not mortality, in

a humanized mouse model of systemic lupus

erythematosus. J Immunol 2001; 167(7):

4083-90.

1997-072 Mouse expressing HLA-DQ6

for the study of rheumatoid

arthritis

These mice express HLA-DQ6

in a mouse class II-deficient

background.

Model system to study

rheumatoid arthritis.

Raju R, et al. T cell recognition of human

pre-proinsulin peptides depends on the

polymorphism at HLA DQ locus: a study using

HLA DQ8 and DQ6 transgenic mice. Hum

Immunol 1997;58(1):21-9.



Adv Immunol 2008;97:65-147.

2000-064 Mouse line expressing human

HLA-DR3 for the study of

autoimmune conditions

These mice express human

HLA-DR3 and are deficient for

the functional mouse H-2 class II

molecules.

Model of autoimmune

conditions, including thyroiditis.

Abraham RS, David CS. Characterization and

kinetic analysis of spontaneous insulitis in

HLA DR3/DQ8 transgenic mice. Hum Immunol

1999;60(Suppl 2): S87.

Das P, et al. HLA transgenic mice as models

of human autoimmune diseases. Rev

Immunogenet 2000;2(1):105-14.



Adv Immunol 2008;97:65-147.

2000-065 Mouse line expressing human

HLA-DQ8 for the study of

rheumatoid arthritis


HLA-DQ8 and are deficient for

the functional mouse H-2 class II

molecules.

Model system to identify

therapeutics for the prevention

and treatment of rheumatoid

arthritis.

Neeno T, et al. HLA-DQ8 transgenic mice

lacking endogenous class II molecules

respond to house dust allergens:

identification of antigenic epitopes. J Immunol

1996;156(9):3191-5.



Adv Immunol 2008;97:65-147.


transgenic mice (cancer models)mayo case # description genotype/phenotype Utility pUblications

1997-112 Preclinical mouse model for

use in MUC1 immunotherapy

protocols

This inbred mouse strain expresses

the human mucin glycoprotein

MUC1 in a tissue-specific fashion

similar to that seen in humans.

Preclinical model for the

study of MUC1, which is

overexpressed on many

epithelial tumors, and MUC1

immunotherapy protocols.

Rowse GJ, et al. Tolerance and immunity in

MUC1 in a human MUC1 transgenic murine

model. Cancer Research 1998;58(2):315-21.

2003-222 Mouse expressing mutant

protein kinase C iota for

the study of colon cancer

progression

Mice were engineered to express

either constitutively active or

kinase-deficient PKCiota in

the colonic epithelium. They

are significantly more or less

susceptible to carcinogen-induced

colon cancer, respectively.

PKCiota active mice develop

adenocarcinomas predominantly

whereas wild-type mice develop

predominantly adenomas.

Model for studying colon cancer

progression.

Murray NR, et al. Protein kinase C iota

is required for Ras transformation and

colon carcinogenesis in vivo. J Cell Biol

2004;164(6):797-802.

2007-110 MUC1-expressing pancreatic

cancer mice

Mice expressing human MUC1

(mucin) were mated with mice

expressing elastase-driven SV40

T antigen. The resulting mice

spontaneously develop pancreatic

tumors that express high levels of

the MUC1.

Useful for investigating immune

responses during tumor

progression.

Mukherjee P, et al. Mice with spontaneous

pancreatic cancer naturally develop MUC-

1-specific CTLs that eradicate tumors

when adoptively transferred. J Immunol

2000;165(6):3451-60.

2008-035 53BP1 knockout mouse model

for the study of DNA damage

and tumor suppression

Mice lack expression of p53

binding protein 1 (53BP1). Mice are

growth retarded, immune deficient,

radiation sensitive, and cancer

prone.

Useful for the study of DNA

damage responses and tumor

suppression.

Ward IM, et al. p53 binding protein 53BP1

is required for DNA damage responses and

tumor suppression in mice. Mol Cell Biol

2003;23(7):2556-2563.

2013-058 Mouse model of multiple

myeloma

Vk*MYC mice have conditional

MYC activation in germinal center

B cells. Mice progress to indolent

myeloma with features highly

characteristic of human disease.

These mice can be used to

study the biology and therapy of

multiple myeloma.

Chesi M, et al. Drug response in a genetically

engineered mouse model of multiple

myeloma is predictive of clinical efficacy.

Blood 2012;120(2):376-85.

Chesi M, et al. AID-dependent activation of

a MYC transgene induces multiple myeloma

in a conditional mouse model of post-

germinal center malignancies. Cancer Cell

2008;13(2):167-80.


transgenic mice (cardiovascular biology and disease models)mayo case # description genotype/phenotype utility publications

2000-020 Mouse strain allowing inducible

gene expression in the heart

Utilizes a tetracycline- inducible

cardiac-specific promoter (α-MHC)

to drive a transactivator activating

expression of any target gene.

System for modulating gene

expression in cardiac tissue.

Valencik ML, McDonald JA. Optimizing

doxycycline regulated gene expression in the

mouse heart. Molecular Biology of the Cell

2000;11(Suppl):129a.

Valencik ML, McDonald JA. Codon

optimization markedly improves doxycycline

regulated gene expression in the mouse

heart. Transgenic research 2001;10(3):269-

275.

2010-040 Mouse overexpressing PAPP-A

in arterial smooth muscle for

the study of atherosclerotic

lesion development

A modified SM22alpha promoter

with a deleted repressor

element drives smooth

muscle overexpression. Upon

crossbreeding with ApoE knockout

mice, PAPP-A transgenic mice

develop larger atherosclerotic

lesions than ApoE knockout mice.


of PAPP-A in cardiovascular

disease.

Conover CA, et al. Transgenic overexpression

of pregnancy-associated plasma protein-A in

murine arterial smooth muscle accelerates

atherosclerotic lesion development. Am J

Physiol Heart Circ Physiol 2010;299(2):H284-

91.

2012-122 Model for studying the role of

TFPI in vascular disease

Mice express murine tissue factor

pathway inhibitor (TFPI) in smooth

muscle cells. TFPI expression

attenuated thrombosis in a ferric

chloride induction model.

Study of vascular disease. Pan S, et al. The effect of vascular smooth

muscle cell-targeted expression of tissue

factor pathway inhibitor in a murine model

of arterial thrombosis. Thromb Haemost

2004;92(3):495-502.

2012-123 Model for studying the role of

TFPI in vascular disease and

other conditions

Mice contain a tissue factor

pathway inhibitor (TFPI) flox allele

with loxP sites flanking exon 4 of

the TFPI gene.

Useful for generating tissue-

specific TFPI deletion and

studying the role of TFPI in

various tissues and diseases.


transgenic mice (immune response models)mayo case # description genotype/phenotype utility publications

1997-071 Mouse expressing HLA-DR4

for studies of CD4-MHC class II

interaction

Key residues in HLA-DR4 were

modified to resemble mouse

sequences, and mice were able to

overcome the species barrier.

Model for the study of MHC

class II binding to CD4.

Pan S, et al. HLA-DR4 (DRB1*0401) transgenic

mice expressing an altered CD4-binding state:

specificity and magnitude of DR4-restricted T

cell response. J Immunol 1998;161(16):2925-

9.

2005-316 Ly9 (CD229) knockout mouse These mice lack expression of the

SLAM family member Ly9. Mice

exhibit various T cell defects.

System for examining Ly9 and

the role in T cell activation.

Graham DB, et al. Ly9 (CD229)-deficient mice

exhibit T cell defects yet do not share several

phenotypic characteristics associated with

SLAM- and SAP-deficient mice. J Immunol

2006;176:291-300.

2006-116 Mice expressing multiple

human HLA class II molecules

for the study of immune

response to bacterial

superantigens

Mice express multiple varieties of

human HLA class II molecules.

Model systems for studying

bacterial superantigens.

Rajagopalan G, et al. Intranasal exposure

to bacterial superantigens induces airway

inflammation in HLA class II transgenic mice.

Infect Immun 2006;74(2):1284-96.


mice mimic human inflammatory diseases. Adv

Immunol 2008;97:65-147.

Rajagopalan G, et al. Evaluating the role of

HLA-DQ polymorphisms on immune response

to bacterial superantigens using transgenic

mice. Tissue Antigens 2008;71(2):135-45.

2010-142 Conditional NKAP knockout

mouse

NKAP is a transcriptional repressor

of the Notch signaling pathway.

The cre-lox system was used to

remove exon 3 of NKAP and disrupt

repressor function of NKAP. Loss

of NKAP function early in T cell

development resulted in a severe

block in T cell development.

Mouse model for examining

NKAP function and the role of

NKAP in T cell development.

Pajerowski AG, et al. NKAP is a transcriptional

repressor of notch signaling and is required for

T cell development. Immunity 2009;30(5):696-

707.


transgenic mice (infectious disease models)mayo case # description genotype/phenotype utility publications

2004-022 Mouse expressing human

CD46 for the study of infectious

disease

Many pathogenic bacteria use

CD46 as a receptor. These mice

express human CD46 with human-

like tissue specificity.

Model for the study of viral

and bacterial infection.

Mrkic B, et al. Lymphatic dissemination and

comparative pathology of recombinant measles

viruses in genetically modified mice. J Virol

2000;74:1364-72.

Mrkic B, et al. Measles virus spread and

pathogenesis in genetically modified mice. J

Virol 1998;72(9):7420-7.

2004-158 Mouse expressing human HGF

as a model of HCV infection


hepatocyte growth factor (HGF)

and support transplanted human

hepatocytes for HCV infection.

Mouse model of HCV infection.

2006-116 Mice expressing multiple

human HLA class II molecules

for the study of immune

response to bacterial

superantigens

Mice express multiple varieties of

human HLA class II molecules.

Model systems for studying

bacterial superantigens.

Rajagopalan G, et al. Intranasal exposure

to bacterial superantigens induces airway

inflammation in HLA class II transgenic mice.

Infect Immun 2006;74(2):1284-96.



Immunol 2008;97:65-147.

Rajagopalan G, et al. Evaluating the role of

HLA-DQ polymorphisms on immune response

to bacterial superantigens using transgenic

mice. Tissue Antigens 2008;71(2):135-45.


transgenic mice (inflammatory disease models)mayo case # description genotype/phenotype Utility pUblications

1997-070 HLA-B27 transgenic mouse

for the study of inflammatory

conditions

Spontaneous inflammatory arthritis

occurs in these HLA-B27 mice

lacking beta 2-microglobulin.

Study of inflammatory

conditions including

spondyloarthropathies.

Khare SD, et al. Spontaneous inflammatory

arthritis in HLA-B27 transgenic mice

lacking beta 2-microglobulin: a model of

human spondyloarthropathies. J Exp Med

1995;182(4):1153-8.



Immunol 2008;97:65-147.

2003-196 Mouse line (PHIL) devoid of

eosinophils for the study of

eosinophils and their roles in

diseases

Eosinophil-specific regulatory

sequences were used in

conjunction with the diphtheria

toxin A gene to eliminate

production of eosinophils.

Model for examining the

role of eosinophils in various

diseases.

Lee JJ, et al. Defining a link with asthma in

mice congenitally deficient in eosinophils.

Science 2004;305(5691):1773-6.

Jacobsen EA, et al. Allergic pulmonary

inflammation in mice is dependent on

eosinophil-induced recruitment of effector T

cells. J Exp Med 2008;205(3):699-710.

2006-291 Eotaxin-2 / IL-5 double

transgenic mouse for the

study of obstructive pulmonary

diseases

Mice express IL-5 systemically

from mature T cells and

eotaxin-2 locally from lung

epithelial cells. These mice

develop several pulmonary

pathologies representative of

obstructive pulmonary disease.

These pulmonary pathologies

are associated with extensive

eosinophil degranulation.

Model of obstructive

pulmonary diseases,

particularly those involving

eosinophils.

Ochkur SI, et al. Coexpression of IL-5 and

eotaxin-2 in mice creates an eosinophil-

dependent model of respiratory inflammation

with characteristics of severe asthma. J

Immunol 2007;178(12):7879-89.

2010-196 iPhil mouse line permitting

inducible loss of eosinophils

to study eosinophil-related

diseases

Inducible ablation of eosinophils is

accomplished through expression

of the human diptheria toxin

receptor exclusively in cells of the

eosinophil lineage. Diptheria toxin

exposure completely eliminates

circulating eosinophils without

affecting other cell types. Depletion

is reversible in days.


of eosinophil recruitment

and activation in allergy,

gastrointestinal disease,

cardiovascular disease, and

transplant rejection.


transgenic mice (metabolic disease models)mayo case # description genotype/phenotype Utility pUblications

2007-230 Human IAPP/amylin knock-in

mouse for the study of amyloid

formation

The mouse islet amyloid

polypeptide (IAPP) gene was

replaced with the human gene.

Mice exhibit symptoms of diabetes

consistent with islet beta cell

dysfunction.

Model to study amyloid

formation and screen

inhibitors for diseases

including diabetes.

2011-030 DBC1 knockout mouse for the

study of metabolic diseases

DBC1 knockout mice have

increased SIRT1 activity in many

tissues including the liver. SIRT1 is

known to be an important regulator

of many biological functions

including energy metabolism.

Deficient mice were protected from

high fat diet-induced liver steatosis

and inflammation despite the

development of obesity.

Useful model system for

the study of DBC1/SIRT1

regulation and the role in

metabolic diseases including

liver steatosis.

Escande C, et al. Deleted in breast cancer-1

regulates SIRT1 activity and contributes to

high-fat diet-induced liver steatosis in mice. J

Clin Invest 2010;120(2):546-558.


transgenic mice (neurodegenerative disease models)mayo case # description genotype/phenotype utility publications

1996-076 Mouse (Tg2576)

overexpressing human Abeta

protein for studying Alzheimer's

disease

These mice overexpress the 695

amino acid form of human Abeta

precursor protein containing

familial AD mutations K670N/

M671L. Mice have increased Abeta

levels accompanied by Abeta

plaques in the brain and behavioral

defects.

Mouse model of Alzheimer's

disease.

Hsiao K, et al. Correlative memory deficits,

Abeta elevation, and amyloid plaques in

transgenic mice. Science 1996;274(5284):99-

102.

2001-023 Mice expressing high levels

of Abeta40 or Abeta42 for

studying amyloid deposition in

Alzheimer's disease

These mice express Abeta40 or

Abeta42 in the secretory pathway.

Sequences encoding Abeta40 or

Abeta42 replace the 23 amino acid

ABri peptide at the C-terminus

of the type II transmembrane

protein BRI resulting in high level

expression of the encoded Abeta

peptide.

Model system for examining

the role of Abeta40 and

Abeta42 in disease.

McGowan E, et al. Abeta42 is essential for

parenchymal and vascular amyloid deposition

in mice. Neuron 2005;47(2):191-9.

Levites Y, et al. Insights into the mechanisms

of action of anti-Abeta antibodies in

Alzheimer's disease mouse models. FASEB J

2006;20(14):2576-8.

Kim J, et al. Abeta40 inhibits amyloid

deposition in vivo. J Neuroscience

2007;27(3):627-33.

2005-016 Mouse with regulated tau

expression as a model of

tauopathy

These transgenic mice have

regulatable tau expression and

profound neurofibrillary pathology,

neurodegeneration and memory

impairment. The protein expressed

is human tau P301L. Transgenic

tau can be suppressed by

administration of the tetracycline

analog, doxycycline.

Model of tauopathies. Santacruz K, et al. Tau suppression

in a neurodegenerative mouse model

improves memory function. Science

2005;309(5733):476-81.

2007-016 Mouse expressing the human

alpha-synuclein gene for the

study of Parkinson's disease

These mice express the human

alpha-synuclein gene, under the

control of its endogenous human

promoter and regulatory elements.

Genomic multiplication of this locus

in humans results in Parkinson's

disease with subsequent dementia,

with post-mortem transitional or

diffuse Lewy body pathology.

Model system for studying

the role of alpha-synuclein in

Parkinson's disease.

2007-027 Mice expressing the human

wild-type and mutant leucine-

rich repeat kinase 2 gene

(LRRK2)

These mice express wild-type or

multiple mutated forms of Lrrk2.

Model of Parkinson's disease. Melrose HL, et al. A comparative analysis of

leucine-rich repeat kinase 2 (Lrrk2) expression

in mouse brain and Lewy body disease.

Neuroscience 2007;147(4):1047-58.


transgenic mice (neurodegenerative disease models)mayo case # description genotype/phenotype utility publications

2007-119 Mouse expressing

human TDP-43 as a

model of dementia

TAR DNA binding protein (TDP-43) is a major

protein constituent of inclusions present in

patients with neurodegenerative diseases.

Model system for studying TDP-43

and human neurodegenerative

diseases including frontotemporal

lobar degeneration and

amyotrophic lateral sclerosis (ALS).

2007-120 Mouse expressing mTau

as a transgenic model of

dementia

Mice form insoluble tau aggregates and

exhibit neuronal pathology.

Model for studying the

mechanisms of tau pathogenesis

and analyzing potential therapeutic

targets.

Adams SJ, et al. Overexpression of wild-

type murine tau results in progressive

tauopathy and neurodegeneration. Am J

Pathol 2009;175(4):1598-609.

2007-288 Leucine-rich repeat

kinase 2 gene (LRRK2)

knockout mouse for the

study of Parkinson's

disease

These mice lack expression of the Lrrk2

protein involved in Parkinson's disease.

Model for assessing the role of

Lrrk2 in Parkinson's disease.

2007-330 LRRK2 wild-type and

mutant (G2019S)

transgenic mice

Lrrk2 expression is under the control of a

tetracycline operator.



2008-163 Inducible tau mouse

expressing human 4RON

tau with the P301L

mutation

These mice conditionally express human

tau (4RON isoform) containing the FTDP-17

mutation (P301L) in exon 10. These mice

develop pre-tangle pathology and neuronal

loss.

Model of tauopathy. Santacruz K, et al. Tau suppression

in a neurodegenerative mouse model

improves memory function. Science

2005;309(5733):476-81.

2012-201 LRRK2 mutant (G2019S)

knock-in mouse

The G2019S pathogenic amino acid

substitution has been incorporated into the

endogenous murine leucine-rich repeat kinase

2 (LRRK2) gene. Mice have normal expression

levels and within 12 months have reduced

release of extracellular dopamine and changes

in monoamine metabolism.



2009-126 Mouse with inducible

expression of human

TDP-43 (iTDP-43WT) as

a model of dementia

TAR DNA binding protein (TDP-43) is a major

protein constituent of inclusions present in

patients with neurodegenerative diseases.

These mice conditionally express human

TDP-43 under a CamKII promoter within

the forebrain in a tetracycline-dependent

manner and induction of expression results in

progressive neurodegeneration.






2012-084 TDP-43 mutant (M377V)

mouse

Mice constitutively express human TDP-

43 (M377V). Mouse TDP-43 expression is

decreased considerably. Mice exhibit reactive

gliosis, motor dysfunction, and early lethality.






Xu, Y et al. Expression of mutant

TDP-43 induces neuronal dysfunction

in transgenic mice. Mol Neurodegener

2011;6:73.


transgenic mice (miscellaneous mouse models)mayo case # description genotype/phenotype utility publications

2003-108 TGF-beta inducible early gene

(TIEG) knockout mouse

These mice might have defects

in osteoblast and osteoclast

differentiation and develop left

ventricular hypertrophy.

Model for studying the

role of TIEG in bone

formation, cardiac myocyte

differentiation, cell

proliferation, cancer, and

connective tissue in addition to

hypertrophic cardiomyopathy.

Subramaniam M, et al. TIEG1 null mouse-

derived osteoblasts are defective in

mineralization and in support of osteoclast

differentiation in vitro. Mol Cell Biol

2005;25(3):1191-9.

Bensamoun SF, et al. TGFbeta inducible early

gene-1 knockout mice display defects in

bone strength and microarchitecture. Bone

2006;39(6):1244-51.

2003-150 Immediate early gene IEX-1

knockout mouse model of

hypertension

These mice have a mean

arterial blood pressure that is

approximately 25mmHg higher

than that seen in wild type mice.

These mice are useful for

studying hypertension and

assessing compounds for the

ability to reduce high blood

pressure.

Sommer SL, et al. Elevated blood pressure and

cardiac hypertrophy after ablation of the gly96/

IEX-1 gene. J Appl Physiol 2006;100(2):707-16.

2004-108 FKBP51 knockout mouse These mice do not express the

immunophilin FKBP51.

Study of the immunophilin

FKBP51 and immunophilin

ligands.

2004-109 FKBP52 knockout mouse These mice do not express the

immunophilin FKBP52.

Study of immunophilin

FKBP52 and immunophilin

ligands.

Cheung-Flynn J, et al. Physiological role for

the cochaperone FKBP52 in androgen receptor

signaling. Mol Endocrinol 2005;19(6):1654-66.

2009-216 Mouse expressing human rab9

GTPase

Mice demonstrate a dramatic

reduction in storage of gangliosides

and an approximately 22%

increase in lifespan relative to

controls.

Useful for studies of secretion

in the intestine and/or liver.

Possible application to other

lysosomal storage diseases.

Model for in vivo studies of

rab9 regulation and activity.

Kaptzan T, et al. Development of a Rab9

transgenic mouse and its ability to increase the

lifespan of a murine model of niemann-pick

type C disease. Am J Pathol 2009;174:14-20.

2009-296 PKHD1 knockout mouse for

the study of polycystic kidney

disease

Autosomal recessive polycystic

kidney disease is caused by

mutations in the PKHD1 gene.

These mice develop liver and

kidney cysts.

Transgenic model of polycystic

kidney disease to study

disease pathogenesis and

therapy.

Woollard JR, et al. A mouse model of autosomal

recessive polycystic kidney disease with biliary

duct and proximal tubule dilation. Kidney

Internat 2007;72:328-336.

2012-144 Mouse model of autosomal

dominant polycystic kidney

disease

Knock-in mice carry the PKD1

R3277C (RC) mutation. RC/null

mice develop severe, early onset

disease. Model reproduces the

anatomical and physiological

features of human disease.

Study of autosomal dominant

polycystic kidney disease and

potential therapeutics.

Hopp K, et al. Functional polycystin-1 dosage

governs autosomal dominant polycystic

kidney disease severity. J Clin Invest

2012;122(11):4257-73.


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