list of animal models

Selecting the optimal disease model is essential for the success of preclinical studies. This page organizes highly cited, foundational review articles from various fields, along with the latest reviews (as of April 15, 2026). It covers key points such as selection based on evaluation objectives and consideration of translatability. Please use this as a reference for appropriate model selection and study design.

Central nervous system (brain)

Alzheimer's disease

APP/PS1 Tg (B6;C3-Tg(APPswe,PSEN1dE9))

Target disease/phenotype	Amyloid plaques, cognitive impairment
Major CRO	Biospective Taconic Biosciences
Examples of marketed drugs and development candidates	Anti-Aβ antibodies (aducanumab, lecanemab, etc.)

Original paper/Published review

• Mutant presenilins specifically elevate the levels of the 42-amino acid β-amyloid peptide in vivo: evidence for augmentation of a 42-specific γ secretase
  Human Molecular Genetics, 13(2):159–170, 2004

icv-STZ

Target disease/phenotype	Neuroinflammation
Major CRO	SMC Laboratories
Examples of marketed drugs and development candidates	-

Original paper/Published review

• Intracerebroventricular administration of streptozotocin causes long-term diminutions in learning and memory abilities and in cerebral energy metabolism in adult rats
  Behavioural Neuroscience, 112(5):1199–1208, 1998

Parkinson's disease

alpha-synuclein A53T Tg

Target disease/phenotype	Lewy bodies, motor disorder
Major CRO	Biospective Scantox The Jackson Laboratory
Examples of marketed drugs and development candidates	-

Original paper/Published review

• Neuronal alpha-synucleinopathy with severe movement disorder in mice expressing A53T human alpha-synuclein
  Neuron, 34(4):521–533, 2002

Image source: Neuronal α-Synucleinopathy with Severe Movement Disorder in Mice Expressing A53T Human α-Synuclein
(https://www.cell.com/neuron/fulltext/S0896627302006827)

Dravet syndrome

SCN1A^+/-Dravet Model

Target disease/phenotype	Epileptic seizures, developmental delay
Major CRO	The Jackson Laboratory
Examples of marketed drugs and development candidates	Fenfluramine

Original paper/Published review

• Reduced sodium current in GABAergic interneurons in a mouse model of severe myoclonic epilepsy in infancy
  Nature Neuroscience, 9(9):1142–1149, 2006

Multiple sclerosis

EAE (Mog-induced)

Target disease/phenotype	Demyelinating encephalomyelitis
Major CRO	MD Biosciences
Examples of marketed drugs and development candidates	Glatiramer acetate (Copaxone), Rituxan (rituximab)

Original paper/Published review

• A myelin oligodendrocyte glycoprotein peptide induces typical chronic experimental autoimmune encephalomyelitis in H-2b mice
  European Journal of Immunology, 25(7):1951–1959, 1995

Cardiovascular system

Arteriosclerosis

ApoE^−/−DECK

Target disease/phenotype	Arteriosclerosis (hyperlipidemia, plaque formation)
Major CRO	Charles River Taconic Biosciences
Examples of marketed drugs and development candidates	Ezetimibe, statin

Original paper/Published review

• Spontaneous hypercholesterolemia and arterial lesions in mice lacking apolipoprotein E
  Science, 258(5081):468–471, 1992

LDLR^−/−DECK

Target disease/phenotype	Arteriosclerosis (Cholesterol Metabolism Disorder)
Major CRO	The Jackson Laboratory
Examples of marketed drugs and development candidates	PCSK9 inhibitors (Evolocumab, Alirocumab)

Original paper/Published review

• Massive xanthomatosis and atherosclerosis in cholesterol-fed low-density lipoprotein receptor-negative mice
  Journal of Clinical Investigation, 93(5):1885–1893, 1994

Image Source: Massive xanthomatosis and atherosclerosis in cholesterol-fed low-density lipoprotein receptor-negative mice
Journal of Clinical Investigation, 93(5):1885–1893, 1994
(https://pmc.ncbi.nlm.nih.gov/articles/PMC294295/)

High blood pressure

Continuous Angiotensin II Infusion

Target disease/phenotype	High blood pressure, cardiac hypertrophy
Major CRO	-
Examples of marketed drugs and development candidates	-

Original paper/Published review

• Angiotensin II promotes atherosclerotic lesions and aneurysms in apolipoprotein E-deficient mice
  Journal of Clinical Investigation, 105(11):1605–1612, 2000

Image source: Angiotensin II promotes atherosclerotic lesions and aneurysms in apolipoprotein E–deficient mice
The impact of chronic inflammation on the pathogenesis of rheumatoid arthritis

Myocardial infarction

Acute myocardial infarction (LAD ligation)

Target disease/phenotype	Myocardial infarction, decreased cardiac function
Major CRO	-
Examples of marketed drugs and development candidates	SGLT2 inhibitor (dapagliflozin)

Original paper/Published review

• Experimental myocardial infarction. I. A method of coronary occlusion in small animals
  Annals of Surgery, 140(5):675–682, 1954

Image source: Experimental myocardial infarction. I. A method of coronary occlusion in small animals
Annals of Surgery, 140(5):675–682, 1954
(https://pmc.ncbi.nlm.nih.gov/articles/PMC1609650/)

Liver

Humanized liver chimeric mouse

Target disease/phenotype	Human drug metabolism (CYPs, etc.), drug-induced liver injury (DILI), viral infections (HBV/HCV), metabolic liver diseases (NASH/MASH)
Major CRO	-
Examples of marketed drugs and development candidates	Antiviral drugs (HBV/HCV), MASH therapeutic drugs, nucleic acid drugs (siRNA/mRNA), AAV gene therapy

Original paper/Published review

• Generation of humanized liver in mice using wet-lab techniques and its applications
• American Journal of Pathology
• Chimeric mice with humanized liver: a unique tool for predicting drug metabolism, toxicity, and safety in humans
• Current Drug Metabolism
• Advances in humanized mouse models for metabolic liver diseases
• (NASH/MASH Model Review)
• Humanized liver models as predictive tools for RNA therapeutic success
• (Review of Nucleic Acid Drugs and Gene Therapy)

Liver Fibrosis, Liver Cirrhosis

Carbon tetrachloride

Target disease/phenotype	Liver fibrosis/cirrhosis
Major CRO	MELIOR DISCOVERY, INC. SMC Laboratories
Examples of marketed drugs and development candidates	-

Original paper/Published review

• Carbon tetrachloride hepatotoxicity: an example of lethal cleavage
  CRC Critical Reviews in Toxicology, 2(3):263–297, 1973

WD+CCl4

Target disease/phenotype	Liver fibrosis/cirrhosis
Major CRO	SMC Laboratories
Examples of marketed drugs and development candidates	-

Original paper/Published review

• Carbon tetrachloride (CCl4) accelerated the development of non-alcoholic fatty liver disease (NAFLD)/steatohepatitis (NASH) in MS-NASH mice fed a western diet supplemented with fructose (WDF).
  BMC Gastroenterology, 20:339, 2020

Image source: Carbon tetrachloride (CCl4) accelerated development of non-alcoholic fatty liver disease (NAFLD)/steatohepatitis (NASH) in MS-NASH mice fed western diet supplemented with fructose (WDF)
(https://pubmed.ncbi.nlm.nih.gov/33059584/)

TAA

Target disease/phenotype	Liver fibrosis/cirrhosis
Major CRO	SMC Laboratories
Examples of marketed drugs and development candidates	-

Original paper/Published review

• Activation of hepatic stellate cells is associated with cytokine expression in thioacetamide-induced hepatic fibrosis in mice
  Laboratory Investigation, 88(11): 1192–1203, 2008

Image source: Activation of hepatic stellate cells is associated with cytokine expression in thioacetamide-induced hepatic fibrosis in mice
（https://www.laboratoryinvestigation.org/article/S0023-6837%2822%2901834-7/fulltext）

Metabolic dysfunction-associated fatty liver disease / Metabolic dysfunction-associated steatohepatitis (NASH)

MASH is a disease characterized by the continuous progression of lipid metabolism abnormalities, inflammation, and fibrosis, and is an area difficult to replicate with a single model.

• Classic review
  • Animal models of nonalcoholic fatty liver disease/nonalcoholic steatohepatitis World Journal of Gastroenterology (2012)
    (https://pubmed.ncbi.nlm.nih.gov/22654421/)
  • Animal Models of Nonalcoholic Fatty Liver Disease—A Starter's Guide Nutrients (2017)
    (https://pubmed.ncbi.nlm.nih.gov/28953222/)
• Latest reviews
  • Mouse models of nonalcoholic steatohepatitis and their application to new drug development Archives of Pharmacal Research (2022)
    https://pubmed.ncbi.nlm.nih.gov/36318445/
  • Mouse Models for the Study of Liver Fibrosis Regression In Vivo and Ex Vivo Journal of Clinical and Translational Hepatology (2024)
    Dapagliflozin attenuates hyperglycemia-induced inflammation and oxidative stress in NAFLD by inhibiting the MAPK/NF-κB and PI3K/Akt/Nrf2 signaling pathways.

In this field, evaluation by combining diet-induced, toxicity-induced, and genetically modified models is considered important.

High-fat diet (HFD)

Target disease/phenotype	MAFLD/MASLD (Fatty Liver, Inflammation, Early Fibrosis)
Major CRO	Charles River Taconic Biosciences
Examples of marketed drugs and development candidates	Orlistat

Original paper/Published review

• High-fat diets: modeling the metabolic disorders of human obesity in rodents
  Obesity, 15(4):798–808, 2007

CDAHFD food

Target disease/phenotype	MASH model (Metabolic dysfunction-Associated Steatohepatitis)
Major CRO	SMC Laboratories Gubra A/S
Examples of marketed drugs and development candidates	Semaglutide

Original paper/Published review

• An improved mouse model for rapid development of fibrosis in non-alcoholic steatohepatitis
  International Journal of Experimental Pathology, 94(2):93–103, 2013

Image source: An improved mouse model that rapidly develops fibrosis in non-alcoholic steatohepatitis
(https://pmc.ncbi.nlm.nih.gov/articles/PMC3607137/)

STAM (Type 2 Diabetes + MASH)

Target disease/phenotype	T2D with MASH
Major CRO	SMC Laboratories
Examples of marketed drugs and development candidates	Senicriviroc, Pegaparbarmarin

Original paper/Published review

• A murine model for non-alcoholic steatohepatitis showing evidence of association between diabetes and hepatocellular carcinoma
  Medical Molecular Morphology, 46(3):141–152, 2013

Biliary and metabolic liver disease

Models and evaluation metrics used differ significantly between acute liver injury and chronic fibrosis.

• Classic review
  • Mechanisms of Hepatotoxicity Toxicological Sciences (2002)
    ( https://pubmed.ncbi.nlm.nih.gov/11812920/ )
  • Liver fibrosis Journal of Clinical Investigation (2005)
    (https://pubmed.ncbi.nlm.nih.gov/15690074/)
• Latest reviews
  • An update on animal models of liver fibrosis Frontiers in Medicine (2023)
    (https://pubmed.ncbi.nlm.nih.gov/37035335/)
  • Experimental models of liver injury and their clinical relevance Hepatology (2024)
    https://pmc.ncbi.nlm.nih.gov/articles/PMC11960460/

In recent years, the importance of selecting models with translational properties in mind has been emphasized.

Bile duct ligation (BDL)

Target disease/phenotype	Cholestatic liver disease (PBC/PSC)
Major CRO	SMC Laboratories
Examples of marketed drugs and development candidates	Obeticholic acid (OCA)

Original paper/Published review

• Bile duct ligation in mice: induction of inflammatory liver injury and fibrosis by obstructive cholestasis
  Journal of Visualized Experiments, (96):e52438, 2015

Image source: Bile Duct Ligation in Mice: Induction of Inflammatory Liver Injury and Fibrosis by Obstructive Cholestasis
Bile Duct Ligation in Mice as a Model for Inducing Inflammatory Liver Injury and Fibrosis

DDC food / ANIT

※Model showing similar pathological conditions (cholangitis/fibrosis)

Target disease/phenotype	Biliary hepatitis/fibrosis
Major CRO	SMC Laboratories
Examples of marketed drugs and development candidates	-

Original paper/Published review

• A new xenobiotic-induced mouse model of sclerosing cholangitis and biliary fibrosis (DDC)
  American Journal of Pathology, 171(2):525–536, 2007
• Drug and chemical-induced intrahepatic cholestasis (ANIT)
  Pharmacological Reviews, 28(3):207–273, 1976

Image source: A new xenobiotic-induced mouse model of sclerosing cholangitis and biliary fibrosis
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Wilson's Disease Model (Atp7b^−/−)

Target disease/phenotype	Wilson's Disease (Copper Metabolism Disorder, Liver Damage)
Major CRO	-
Examples of marketed drugs and development candidates	-

Original paper/Published review

• Null mutation of the murine ATP7B gene results in copper accumulation and liver disease
  Hum Mol Genet, 8(9):1665-71, 1999

Metabolic and inflammatory hepatocellular carcinoma

Hepatocellular carcinoma (HCC)

STAM-IO (STZ + HFD)

Target disease/phenotype	MASH-HCC under normal immunity (tumor microenvironment with fibrosis; immune response evaluation)
Major CRO	SMC Laboratories
Examples of marketed drugs and development candidates	-

Original paper/Published review

• A murine model for non-alcoholic steatohepatitis showing evidence of association between diabetes and hepatocellular carcinoma
  Medical Molecular Morphology, 46(3):141–152, 2013

CDAHFD-induced HCC model

Target disease/phenotype	Spontaneous hepatocellular carcinoma formation from MASH with severe inflammation and fibrosis
Major CRO	SMC Laboratories Gubra A/S
Examples of marketed drugs and development candidates	-

Original paper/Published review

• An improved mouse model for rapid development of fibrosis in non-alcoholic steatohepatitis
  International Journal of Experimental Pathology, 94(2):93–103, 2013

Image source: An improved mouse model that rapidly develops fibrosis in non-alcoholic steatohepatitis
(https://pmc.ncbi.nlm.nih.gov/articles/PMC3607137/)

Western Diet + CCl₄ Model (WD+CCl₄)

Target disease/phenotype	Progression from NASH with advanced fibrosis to HCC
Major CRO	SMC Laboratories
Examples of marketed drugs and development candidates	-

Original paper/Published review

• Carbon tetrachloride (CCl4) accelerated the development of non-alcoholic fatty liver disease (NAFLD)/steatohepatitis (NASH) in MS-NASH mice fed a western diet supplemented with fructose (WDF).
  BMC Gastroenterology, 20:339, 2020

Image source: Carbon tetrachloride (CCl4) accelerated development of non-alcoholic fatty liver disease (NAFLD)/steatohepatitis (NASH) in MS-NASH mice fed western diet supplemented with fructose (WDF)
(https://pmc.ncbi.nlm.nih.gov/articles/PMC7560288/)

DEN+CCl₄ model

Target disease/phenotype	Fibrotic background HCC (chronic liver injury + chemical carcinogenesis; tumorigenesis with liver cirrhosis)
Major CRO	SMC Laboratories
Examples of marketed drugs and development candidates	-

Original paper/Published review

• The DEN and CCl4-Induced Mouse Model of Fibrosis and Inflammation-Associated Hepatocellular Carcinoma
  Current Protocol, 1(8):e211

Image Source: The DEN and CCl4-induced Mouse Model of Fibrosis and Inflammation-associated Hepatocellular Carcinoma
(https://pmc.ncbi.nlm.nih.gov/articles/PMC4214366/)

Kidney

In kidney disease, surgical models and metabolic models are used in combination.

• Classic review
  • Molecular basis of renal fibrosis Pediatric Nephrology (2000)
    The role of the endocannabinoid system in sleep regulation.
  • Obstructive nephropathy and renal fibrosis American Journal of Physiology-Renal Physiology (2002)
    (https://pubmed.ncbi.nlm.nih.gov/12372761/)
• Latest reviews
  • Obstructive nephropathy and molecular pathophysiology of renal interstitial fibrosis Physiological Reviews (2023)
    (https://pubmed.ncbi.nlm.nih.gov/37440209/)
  • Kidney fibrosis in vitro and in vivo models: path toward physiologically relevant humanized models Advanced Healthcare Materials (2025)
    Title: Exploring the Landscape of AI-Driven Drug Discovery: Promises, Pitfalls, and Future Directions Abstract Artificial intelligence (AI) has rapidly emerged as a transformative force in drug discovery, offering unprecedented opportunities to accelerate the identification, design, and development of novel therapeutics. This review explores the multifaceted landscape of AI-driven drug discovery, examining its promises, inherent pitfalls, and charting a course for its future trajectory. We delve into the various AI methodologies, including machine learning, deep learning, and natural language processing, that are being applied across the drug discovery pipeline, from target identification and validation, hit identification, lead optimization, to preclinical and clinical trial design. The review highlights key successes and groundbreaking applications that demonstrate AI's potential to overcome traditional bottlenecks, reduce costs, and improve success rates. However, the path to AI-driven drug discovery is not without its challenges. We critically assess the pitfalls, including data quality and accessibility issues, the need for robust validation and interpretability of AI models, ethical considerations, regulatory hurdles, and the integration of AI into existing R&D workflows. The "black box" nature of some AI algorithms poses significant challenges for understanding the underlying biological mechanisms and gaining regulatory approval. Furthermore, the rapid evolution of AI techniques necessitates continuous learning and adaptation for researchers and pharmaceutical companies. Looking ahead, this review outlines future directions for AI in drug discovery. We emphasize the importance of developing explainable AI (XAI) methods to foster trust and transparency. The integration of diverse data sources, including genomics, proteomics, metabolomics, and real-world evidence, will be crucial for building more comprehensive and predictive models. The role of AI in personalized medicine, enabling the tailoring of drug therapies to individual patient profiles, will continue to expand. Moreover, fostering interdisciplinary collaboration between AI experts, biologists, chemists, clinicians, and regulatory bodies is paramount for realizing the full potential of AI in revolutionizing drug discovery and development, ultimately leading to faster access to life-saving treatments for patients. Keywords: Artificial intelligence, drug discovery, machine learning, deep learning, natural language processing, R&D, therapeutics, personalized medicine, explainable AI, drug development.

In recent years, the introduction of humanized models and 3D culture systems has been advancing.

Diabetic nephropathy - diabetic model

db/db (Lepr^−/−)

Target disease/phenotype	Type 2 diabetic nephropathy (obesity, hyperglycemia)
Major CRO	The Jackson Laboratory Charles River Laboratories, Inc. Taconic Biosciences
Examples of marketed drugs and development candidates	SGLT2 inhibitors (e.g., dapagliflozin)

Original paper/Published review

• Evidence that the diabetes gene encodes the leptin receptor: identification of a mutation in the leptin receptor gene in db/db mice
  Cell, 84(3):491–495, 1996

Image Source: Evidence That the Diabetes Gene Encodes the Leptin Receptor: Identification of a Mutation in the Leptin Receptor Gene in db/db Mice
（https://www.cell.com/fulltext/S0092-8674%2800%2981294-5）

NOD mouse

Target disease/phenotype	Type 1 diabetes (autoimmune destruction of pancreatic beta cells)
Major CRO	The Jackson Laboratory Taconic Biosciences
Examples of marketed drugs and development candidates	-

Original paper/Published review

• Breeding of a non-obese, diabetic strain of mice
  Experimental Animals, 29(1):1–13, 1980

OVE26 (T1D)

Target disease/phenotype	Type 1 diabetes model (insulin deficiency)
Major CRO	-
Examples of marketed drugs and development candidates	-

Original paper/Published review

• Calmodulin-induced early-onset diabetes in transgenic mice
  Cell, 58(6):1067–1073, 1989

Akita (Ins2^+/-)

Target disease/phenotype	Type 1 Diabetes (Hereditary Insulin Deficiency)
Major CRO	The Jackson Laboratory
Examples of marketed drugs and development candidates	-

Original paper/Published review

• A novel locus, Mody4, distal to D7Mit189, contributes to early-onset NIDDM in nonobese C57BL/6 (Akita) mutant mice
  Diabetes, 46(5):887–894, 1997

eNOS^−/−+ db/db

Target disease/phenotype	Hypertensive diabetic nephropathy
Major CRO	-
Examples of marketed drugs and development candidates	-

Original paper/Published review

• Diabetic endothelial nitric oxide synthase knockout mice develop advanced diabetic nephropathy
  Journal of the American Society of Nephrology, 18(2):539–550, 2007

Cystic kidney disease, hereditary, autoimmune kidney disease

Polycystic kidney disease (Pkd1 KO)

Target disease/phenotype	Autosomal dominant polycystic kidney disease (ADPKD)
Major CRO	The Jackson Laboratory
Examples of marketed drugs and development candidates	Tolvaptan

Original paper/Published review

• Perinatal lethality with kidney and pancreas defects in mice with a targeted Pkd1 mutation
  Nature Genetics, 17(2):179–181, 1997

Alport syndrome (Col4a3^−/−)

Target disease/phenotype	Alport syndrome (abnormal glomerular basement membrane)
Major CRO	The Jackson Laboratory
Examples of marketed drugs and development candidates	-

Original paper/Published review

• Collagen COL4A3 knockout: a mouse model for autosomal Alport syndrome
  Genes & Development, 10(23):2981–2992, 1996

Lupus Nephritis (NZB/NZW F1)

Target disease/phenotype	Systemic lupus erythematosus (nephritis)
Major CRO	The Jackson Laboratory
Examples of marketed drugs and development candidates	-

Original paper/Published review

• Spontaneous autoimmune disease in (NZB x NZW)F1 mice is ameliorated by treatment with methimazole
  J Clin Immunol, 18(2):106-13, 1998

Lung

Asthma/Airway Inflammation

OVA asthma induction model

Target disease/phenotype	Allergic asthma (eosinophilic airway inflammation)
Major CRO	Charles River Laboratories, Inc. Inotiv, Inc.
Examples of marketed drugs and development candidates	Dupilumab

Original paper/Published review

• OVA-Induced Allergic Airway Inflammation Mouse Model
  Methods in Molecular Biology, 1916:297–301, 2019

HDM asthma model

Target disease/phenotype	Allergic airway inflammation
Major CRO	Charles River Laboratories, Inc. Inotiv, Inc.
Examples of marketed drugs and development candidates	-

Original paper/Published review

• Continuous exposure to house dust mites causes chronic inflammation and structural changes in the airways.
  American Journal of Respiratory and Critical Care Medicine, 169(3):378–385, 2004

Pulmonary fibrosis

IPF is a disease area where the balance between reproducibility and clinical correlation is important.

• Classic review
  • Murine Models of Pulmonary Fibrosis American Journal of Physiology-Lung Cellular and Molecular Physiology (2008)
  • The bleomycin animal model: a useful tool to investigate treatment options for idiopathic pulmonary fibrosis? International Journal of Biochemistry & Cell Biology (2008) (https://pubmed.ncbi.nlm.nih.gov/17936056/)
• Latest reviews
  • Effectiveness and mechanism of metformin in animal models of pulmonary fibrosis: A systematic review Frontiers in Pharmacology (2022) (https://pubmed.ncbi.nlm.nih.gov/36147352/)
  • Current advancements in the mechanisms and animal models of pulmonary fibrosis Frontiers in Pharmacology (2025)

In recent years, there has been growing interest in chronic models and humanized models.

Bleomycin-induced pulmonary fibrosis

Target disease/phenotype	Pulmonary fibrosis (interstitial pneumonia)
Major CRO	SMC Laboratories
Examples of marketed drugs and development candidates	Pirfenidone, nintedanib, nelondomilast

Original paper/Published review

• The pathogenesis of bleomycin-induced pulmonary fibrosis
  American Journal of Pathology, 77(2):185–197, 1974

Image source: The Pathogenesis of Bleomycin-Induced Pulmonary Fibrosis in Mice
(https://pmc.ncbi.nlm.nih.gov/articles/PMC1910906/)

Silicosis

Target disease/phenotype	Pulmonary fibrosis (interstitial pneumonia)
Major CRO	SMC Laboratories
Examples of marketed drugs and development candidates	-

Original paper/Published review

• Development of experimental silicosis in inbred and genetically diverse mice
  Scientific Reports, 9:16330, 2019

Image source: Development of experimental silicosis in inbred and outbred mice depends on instillation volume
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Infectious disease

COVID-19 model

Target disease/phenotype	SARS-CoV-2 infection (ARDS-like lung injury)
Major CRO	Charles River Laboratories, Inc. The Jackson Laboratory
Examples of marketed drugs and development candidates	-

Original paper/Published review

• SARS-CoV-2 infection of human ACE2-transgenic mice causes severe lung inflammation and impaired function
  Nature Immunology, 21(11):1327–1335, 2020

Image Source: SARS-CoV-2 infection of human ACE2-transgenic mice causes severe lung inflammation and impaired function
(https://www.nature.com/articles/s41590-020-0778-2)

Gastrointestinal tract

Inflammatory Bowel Disease (IBD)

Inflammation models vary greatly depending on the induction method.

• Classic review
  • Mouse models of inflammatory bowel disease Adv Drug Deliv Rev. (2007)
    (https://pubmed.ncbi.nlm.nih.gov/17446870/)
• Latest reviews
  • Animal models of inflammatory bowel disease: novel experiments for revealing pathogenesis Intestinal Research (2023)
    (https://pubmed.ncbi.nlm.nih.gov/37248173/)
  • Animal Models of Inflammatory Bowel Disease: Classification and Evaluation Indices Gastroenterology Report (2024)
    A role for the 7-gene microRNA-122/microRNA-122-5p targeting 7-gene panel in the differential diagnosis of liver nodules in patients with liver cirrhosis.

In recent years, models combining human gut microbiota have also been attracting attention.

DSS colitis

Target disease/phenotype	Inflammatory bowel disease (Ulcerative colitis)
Major CRO	SMC Laboratories Gubra A/S
Examples of marketed drugs and development candidates	-

Original paper/Published review

• A novel method for inducing experimental ulcerative colitis in mice
  Gastroenterology, 98(3):694–702, 1990

TNBS colitis

Target disease/phenotype	Crohn's disease-like colitis
Major CRO	Charles River Laboratories, Inc. Inotiv, Inc.
Examples of marketed drugs and development candidates	-

Original paper/Published review

• Hapten-induced model of chronic inflammation and ulceration in the rat colon
  Gastroenterology, 96(3):795–803, 1989

IL-10KO colitis

Target disease/phenotype	Spontaneous Colitis (Chronic Inflammation)
Major CRO	The Jackson Laboratory
Examples of marketed drugs and development candidates	-

Original paper/Published review

• Interleukin-10 deficient mice develop chronic enterocolitis.
  Cell, 75(2):263–274, 1993

Stomach and colorectal diseases

Helicobacter pylori infection

Target disease/phenotype	Chronic gastritis
Major CRO	-
Examples of marketed drugs and development candidates	-

Original paper/Published review

• Helicobacter bilis-associated inflammatory bowel disease in defined microbiota mice
  Infection and Immunity, 72(6):3328–3337, 2004

Image Source: Helicobacter bilis Infection Alters Mucosal Bacteria and Modulates Colitis Development in Defined Microbiota Mice
(https://pmc.ncbi.nlm.nih.gov/articles/PMC5123692/)

AOM/DSS Colorectal Cancer

Target disease/phenotype	Colorectal cancer (inflammation-related cancer)
Major CRO	Crown Bioscience Charles River Laboratories, Inc. SMC Laboratories
Examples of marketed drugs and development candidates	-

Original paper/Published review

• Development of an inflammation-associated colorectal cancer model
  Int J Inflam, 658786, 2012

Image Source: Development of an Inflammation-Associated Colorectal Cancer Model and Its Application for Research on Carcinogenesis and Chemoprevention
https://pmc.ncbi.nlm.nih.gov/articles/PMC3299397/

Endocrine and Metabolic System

Obesity-type 2 diabetes

In metabolic diseases, it is important to choose between genetic and dietary models.

• Classic review
  • Animal models of obesity Obesity Reviews (2007)
    (https://pubmed.ncbi.nlm.nih.gov/17316303/)
  • The use of animal models in diabetes research
    (https://pubmed.ncbi.nlm.nih.gov/22352879/)
• Latest reviews
  • Animal Models for Type 1 and Type 2 Diabetes: Advantages and Limitations Frontiers in Endocrinology (2024)
    (https://pubmed.ncbi.nlm.nih.gov/36092796/)
  • Animal Models for Type 1 and Type 2 Diabetes: Advantages and Limitations Frontiers in Endocrinology (2024)
    The impact of AI in health care is enormous.

When selecting models, it is important to consider metabolic differences with humans.

ob/ob^−/−)

Target disease/phenotype	Obesity-type 2 diabetes
Major CRO	The Jackson Laboratory Taconic Biosciences
Examples of marketed drugs and development candidates	-

Original paper/Published review

• Positional cloning of the mouse obese gene and its human homologue
  Nature, 372(6505):425–432, 1994

db/db (Lepr^−/−)

Target disease/phenotype	Obesity-type 2 diabetes
Major CRO	The Jackson Laboratory Charles River Laboratories, Inc.
Examples of marketed drugs and development candidates	Dapagliflozin

Original paper/Published review

• Evidence that the diabetes gene encodes the leptin receptor
  Cell, 84(3):491–495, 1996

Diet-induced obesity (DIO)

Target disease/phenotype	Obesity and metabolic syndrome
Major CRO	Charles River Laboratories, Inc. Taconic Biosciences Gubra A/S SMC Laboratories
Examples of marketed drugs and development candidates	Orlistat

Original paper/Published review

• High-fat diets: modeling the metabolic disorders of human obesity in rodents
  Obesity, 15(4):798–808, 2007

Neonatal STZ model

Target disease/phenotype	Type 2 diabetes (beta-cell dysfunction)
Major CRO	SMC Laboratories
Examples of marketed drugs and development candidates	-

Original paper/Published review

• Studies on the diabetogenic action of streptozotocin
  Cancer Chemotherapy Reports, 29:91–98, 1963

Type 1 diabetes

NOD mouse

Target disease/phenotype	Type 1 diabetes (autoimmune)
Major CRO	The Jackson Laboratory
Examples of marketed drugs and development candidates	-

Original paper/Published review

• Breeding of a non-obese, diabetic strain of mice
  Experimental Animals, 29(1):1–13, 1980

Immune and hematopoietic system

Humanized mice

Target disease/phenotype	In vivo humanized mouse model (T cell response, innate immunity, tumor immunity, immunosuppressive mechanisms)
Major CRO	-
Examples of marketed drugs and development candidates	Immune checkpoint inhibitors, CAR T-cell therapy, bispecific antibodies

Original paper/Published review

• Humanized mice in cancer immunotherapy
• Nature Reviews Cancer
• Humanized mouse models for the study of human immunology and personalized medicine
• Annual Review of Immunology
• Myeloid-enhanced humanized mouse models: advances and challenges
• (Cytokine-Inducing HIS Review)
• Integrating Humanized Mouse Models with Spatial Omics and AI
• (Spatial Omics × AI Integrated Review)

Autoimmune disease

NZB/NZW F1 (SLE)

Target disease/phenotype	Systemic lupus erythematosus (lupus nephritis, etc.)
Major CRO	The Jackson Laboratory
Examples of marketed drugs and development candidates	-

Original paper/Published review

• Spontaneous autoimmune disease in NZB/NZW mice
  British Journal of Haematology, 119-31, 1963

MRL/lpr (SLE)

Target disease/phenotype	SLE (Lymphadenopathy, Cutaneous Renal Lesions)
Major CRO	The Jackson Laboratory
Examples of marketed drugs and development candidates	-

Original paper/Published review

• Lymphoproliferation disorder in mice explained by defects in Fas antigen
  Nature, 356(6367):314–317, 1992

RA (Rheumatoid Arthritis)

Target disease/phenotype	Rheumatoid arthritis model (synovitis, bone destruction)
Major CRO	Inotiv, Inc. Charles River Laboratories, Inc.
Examples of marketed drugs and development candidates	-

Original paper/Published review

• Autoimmunity to type II collagen: an experimental model of arthritis
  Journal of Experimental Medicine, 146(3):857–868, 1977

K/BxN CAIA

Target disease/phenotype	Autoimmune arthritis (antibody-mediated)
Major CRO	-
Examples of marketed drugs and development candidates	-

Original paper/Published review

• Organ-specific autoimmune disease
  Cell, 87(5):811–822, 1996

Image Source: Organ-Specific Disease Provoked by Systemic Autoimmunity
（https://www.cell.com/cell/fulltext/S0092-8674(00)81989-3?_returnURL=https%3A%2F%2Flinkinghub.elsevier.com%2Fretrieve%2Fpii%2FS0092867400819893%3Fshowall%3Dtrue#FIG2）

Immunodeficiency model

NSG (NOD/SCID/γc)^−/−)

Target disease/phenotype	Severe Combined Immunodeficiency (T/B/NK cell deficiency)
Major CRO	The Jackson Laboratory
Examples of marketed drugs and development candidates	-

Original paper/Published review

• NOD/SCID/γcnull mouse: an excellent recipient mouse model for engraftment of human cells
  Blood, 100(9):3175–3182, 2002

Image source: NOD/SCID/γcnull mouse: an excellent recipient mouse model for engraftment of human cells
(https://www.sciencedirect.com/science/article/pii/S0006497120514729)

SCID (CB-17)

Target disease/phenotype	Severe combined immunodeficiency
Major CRO	Charles River Laboratories, Inc. Taconic Biosciences
Examples of marketed drugs and development candidates	-

Original paper/Published review

• A severe combined immunodeficiency mutation in mice
  Nature, 301(5900):527–530, 1983

Musculoskeletal system

Muscular dystrophy, myopathy

mdx (DMD)

Target disease/phenotype	Duchenne muscular dystrophy
Major CRO	CLEA Japan SMC Laboratories
Examples of marketed drugs and development candidates	-

Original paper/Published review

• X chromosome-linked muscular dystrophy (mdx) in the mouse
  Proceedings of the National Academy of Sciences USA, 81(4):1189–1192, 1984

Image source: X chromosome-linked muscular dystrophy (mdx) in the mouse.
(https://pmc.ncbi.nlm.nih.gov/articles/PMC344791/)

D2.mdx (Severe DMD)

Target disease/phenotype	Severe DMD
Major CRO	The Jackson Laboratory
Examples of marketed drugs and development candidates	-

Original paper/Published review

• Genetic background affects satellite cell properties and mdx phenotypes
  American Journal of Pathology, 176(5):2414–2424, 2010

Image source: Genetic background affects properties of satellite cells and mdx phenotypes
(https://pubmed.ncbi.nlm.nih.gov/20304955/)

COL6 deficiency disorder

Target disease/phenotype	Congenital muscular dystrophy
Major CRO	-
Examples of marketed drugs and development candidates	-

Original paper/Published review

• Collagen VI deficiency induces early onset myopathy in the mouse
  Proceedings of the National Academy of Sciences USA, 95(3):1046–1051, 1998

Bone disease

Osteoporosis (OVX)

Target disease/phenotype	Estrogen deficiency osteoporosis
Major CRO	Charles River Laboratories, Inc. Inotiv, Inc.
Examples of marketed drugs and development candidates	-

Original paper/Published review

• The ovariectomized rat model of postmenopausal bone loss
  Bone and Mineral, 15(3):175–191, 1991

Dermatology

Psoriasis/Atopic Dermatitis

Imiquimod model

Target disease/phenotype	Psoriasiform dermatitis (erythema, scaling)
Major CRO	Charles River Laboratories, Inc. Inotiv, Inc. SMC Laboratories
Examples of marketed drugs and development candidates	-

Original paper/Published review

• Imiquimod-induced psoriasis-like skin inflammation
  Journal of Immunology, 182(9):5836–5845, 2009

North Carolina / Nigeria

Target disease/phenotype	Atopic dermatitis (pruritic eczema, elevated IgE)
Major CRO	Charles River Laboratories, Inc.
Examples of marketed drugs and development candidates	-

Original paper/Published review

• NC/Nga mice: a model for atopic dermatitis
  Int Arch Allergy Immunol, 120(Suppl 1):70–75, 1999

Cutaneous fibrosis, contact dermatitis

Bleomycin-induced pulmonary fibrosis

Target disease/phenotype	Systemic sclerosis (SSc)
Major CRO	SMC Laboratories
Examples of marketed drugs and development candidates	-

Original paper/Published review

• Animal model of sclerotic skin. I: Local injections of bleomycin induce sclerotic skin mimicking scleroderma
  Journal of Investigative Dermatology, 112(4):456–462, 1999

Image Source: Animal Model of Sclerotic Skin. I: Local Injections of Bleomycin Induce Sclerotic Skin Mimicking Scleroderma
(https://www.jidonline.org/article/S0022-202X(15)40449-X/pdf)

Contact dermatitis (DNFB)

Target disease/phenotype	Contact dermatitis (allergic inflammation)
Major CRO	Inotiv, Inc.
Examples of marketed drugs and development candidates	-

Original paper/Published review

• A hapten-induced model of allergic contact dermatitis characterized by T cell–mediated skin inflammation following sensitization and challenge with DNFB.

Reproductive and Endocrine Systems

Polycystic Ovary Syndrome (PCOS)

DHEA model (PCOS)

Target disease/phenotype	Polycystic Ovary Syndrome (PCOS)
Major CRO	-
Examples of marketed drugs and development candidates	-

Original paper/Published review

• Effect of dehydroepiandrosterone and Δ4-androstenedione on the reproductive organs of female rats: induction of cystic changes in the ovary
  Nature, 1962;196:42–43

Letrozole model (PCOS)

Target disease/phenotype	PCOS
Major CRO	-
Examples of marketed drugs and development candidates	-

Original paper/Published review

• Letrozole-induced polycystic ovaries in the rat: a new model for cystic ovarian disease.
  Archives of Medical Research, 2004;35(2):103–108.

Uterine and gonadal diseases

Endometriosis model

Target disease/phenotype	Endometriosis
Major CRO	-
Examples of marketed drugs and development candidates	-

Original paper/Published review

• Studies on the Surgical Induction of Endometriosis in the Rat
  Fertility and Sterility, 1985;44(5):684–694.

GnRH receptor KO

Target disease/phenotype	Hypogonadism (amenorrhea, infertility)
Major CRO	-
Examples of marketed drugs and development candidates	-

Original paper/Published review

• Mouse model of surgically induced endometriosis by autotransplantation of uterine tissue.
  Journal of Visualized Experiments, 2012;(59):e3396

Sensory organs, development, and aging

Sensory organ disorders (visual/auditory)

rd1 (Pde6b^rd1)

Target disease/phenotype	Retinitis pigmentosa (blindness)
Major CRO	The Jackson Laboratory
Examples of marketed drugs and development candidates	-

Original paper/Published review

• Mutations in the GnRH receptor gene: a new cause of autosomal recessive hypogonadotropic hypogonadism.
  Archives of Medical Research, 1999;30(6):481–485.

Cx26 KO

Target disease/phenotype	Hereditary hearing loss
Major CRO	-
Examples of marketed drugs and development candidates	-

Original paper/Published review

• Targeted ablation of connexin26 in the inner ear epithelial gap junction network causes hearing impairment and cell death.
  Current Biology, 2002;12(13):1106–1111.

Image Source: Targeted Ablation of Connexin26 in the Inner Ear Epithelial Gap Junction Network Causes Hearing Impairment and Cell Death
(https://pmc.ncbi.nlm.nih.gov/articles/PMC4030438/)

Development, aging, and rare diseases

Ames dwarf (Prop1)^df/df)

Target disease/phenotype	Laron Syndrome Model (GH Deficiency)
Major CRO	The Jackson Laboratory
Examples of marketed drugs and development candidates	-

Original paper/Published review

• Genotyping the Prop-1 mutation in Ames dwarf mice.
  Mechanisms of Ageing and Development, 2001;122(15):1915–1918.

Zmpste24^−/−HGPS

Target disease/phenotype	Hutchinson-Gilford syndrome (progeria)
Major CRO	-
Examples of marketed drugs and development candidates	-

Original paper/Published review

• Zmpste24 deficiency in mice causes spontaneous bone fractures, muscle weakness, and a prelamin A processing defect.
  Proceedings of the National Academy of Sciences USA, 2002;99(20):13049–13054.

Image source: Zmpste24 deficiency in mice causes spontaneous bone fractures, muscle weakness, and a prelamin A processing defect
The patient was a 61-year-old man who had been diagnosed with an obstructing colonic tumor. He underwent a hemicolectomy and then received adjuvant chemotherapy with capecitabine. After 12 cycles of chemotherapy, he experienced recurrent vomiting. Colonoscopy revealed ulceration and stenosis of the sigmoid colon. Biopsy results showed severe submucosal inflammation with eosinophilic infiltration, which was ultimately diagnosed as eosinophilic colitis. The patient responded well to corticosteroid therapy. To our knowledge, this is the first case report of eosinophilic colitis occurring as a complication of capecitabine therapy.

Werner (Wrn^−/−)

Target disease/phenotype	Werner syndrome (progeria)
Major CRO	-
Examples of marketed drugs and development candidates	-

Original paper/Published review

• Metabolic and Phenotypic Differences Between Mice Producing a Werner Syndrome Helicase Mutant Protein and Wrn Null Mice.
  PLoS ONE, 2015;10(10):e0140292.

Image Source: Metabolic and Phenotypic Differences between Mice Producing a Werner Syndrome Helicase Mutant Protein and Wrn Null Mice
(https://pmc.ncbi.nlm.nih.gov/articles/PMC4598085/)

Oncology (Cancer Models)

Tumor models are selected according to the purpose of evaluation.

• Classic review
  • Preclinical mouse cancer models: a maze of opportunities and challenges Cell (2015)
    (https://pubmed.ncbi.nlm.nih.gov/26406370/)
• Latest reviews

• Patient-Derived Xenograft Models in Cancer: Establishment and Applications MedComm (2025)
  https://pubmed.ncbi.nlm.nih.gov/39830019/
• Patient-derived xenograft models: current status, challenges, and innovations Genes & Diseases (2025)
  A study on the immunomodulatory effects of propolis in animal models.

In recent years, the importance of evaluation using PDX and humanized models has been increasing.

PDX model

Target disease/phenotype	Human tumor xenograft models (intratumoral heterogeneity, partial preservation of the tumor microenvironment)
Major CRO	Crown Bioscience Charles River Laboratories, Inc. Champions Oncology, Inc.
Examples of marketed drugs and development candidates	Immune checkpoint inhibitors, molecular targeted drugs (various solid tumor areas)

Original paper/Published review

• Patient-derived tumor xenografts as models for oncology drug development
• Nature Reviews Clinical Oncology
• High-throughput screening using patient-derived tumor xenografts to predict clinical trial drug response
• Nature Medicine
• Recent advances in humanized mouse models for cancer immunotherapy
• (Immunotherapy / Humanized PDX Review)
• Integrating patient-derived models with spatial transcriptomics and AI for precision oncology
• (Spatial Omics × AI Integrated Review)

Digestive cancer, lung cancer

Large intestine: Apc gene deletion

Target disease/phenotype	Colorectal tumor (multiple intestinal tumors)
Major CRO	The Jackson Laboratory
Examples of marketed drugs and development candidates	-

Original paper/Published review

• A dominant mutation that predisposes to multiple intestinal neoplasia in the mouse
  Science, 247(4940):322–324, 1990

Colon: Azoxymethane injection + DSS

Target disease/phenotype	Inflammatory bowel disease-associated colorectal cancer
Major CRO	Crown Bioscience Charles River Laboratories, Inc. SMC Laboratories
Examples of marketed drugs and development candidates	-

Original paper/Published review

• Dextran sodium sulfate strongly promotes colorectal carcinogenesis in ApcMin/+ mice: inflammatory stimuli by dextran sodium sulfate results in development of multiple colonic neoplasms
  International Journal of Cancer, 118(1):25–34, 2006

Lung: Kras^GNDTRANS GENIC INC.

Target disease/phenotype	Lung adenocarcinoma
Major CRO	The Jackson Laboratory
Examples of marketed drugs and development candidates	-

Original paper/Published review

• Analysis of lung tumor initiation and progression using conditional expression of oncogenic K-ras
  Genes & Development, 15(24):3243–3248, 2001

Image Source: Analysis of lung tumor initiation and progression using conditional expression of oncogenic K-ras
(https://pmc.ncbi.nlm.nih.gov/articles/PMC312845/)

Liver cancer, skin cancer, others

Liver: Diethylnitrosamine injection

Target disease/phenotype	Hepatocellular carcinoma (HCC)
Major CRO	SMC Laboratories
Examples of marketed drugs and development candidates	-

Original paper/Published review

• Kinetics of diethylnitrosamine hepatocarcinogenesis in the infant mouse
  Cancer Research, 43(9):4253–4259, 1983

Skin: DMBA application + TPA application

Target disease/phenotype	Squamous papilloma and Squamous cell carcinoma
Major CRO	Charles River Laboratories, Inc.
Examples of marketed drugs and development candidates	-

Original paper/Published review

• The role of croton oil applications associated with a single painting of a carcinogen in tumor induction of the mouse's skin
  British Journal of Cancer, 1(4):383–391, 1947

Breast: Polyoma Middle T Antigen

Target disease/phenotype	Breast cancer (metastasis model)
Major CRO	The Jackson Laboratory
Examples of marketed drugs and development candidates	-

Original paper/Published review

• Induction of mammary tumors by expression of polyomavirus middle T oncogene: a transgenic mouse model for metastatic disease
  Molecular and Cellular Biology, 12(3):954–961, 1992

Prostate: SV40 T antigen (prostate specific)

Target disease/phenotype	Prostate cancer
Major CRO	The Jackson Laboratory
Examples of marketed drugs and development candidates	-

Original paper/Published review

• Prostate cancer in a transgenic mouse
  Proceedings of the National Academy of Sciences USA, 92(8):3439–3443, 1995

Image source: Prostate cancer in a transgenic mouse.
(https://pmc.ncbi.nlm.nih.gov/articles/PMC42182/)

Blood: BCR-ABL fusion gene introduction

Target disease/phenotype	Chronic myeloid leukemia (CML)
Major CRO	Crown Bioscience
Examples of marketed drugs and development candidates	Imatinib

Original paper/Published review

• Induction of chronic myelogenous leukemia in mice by the P210 bcr/abl gene of the Philadelphia chromosome
  Science, 247(4944):824–830, 1990

Skin (Melanoma): B16 melanoma cell injection

Target disease/phenotype	Melanoma (Allogeneic Transplantation Model)
Major CRO	Crown Bioscience
Examples of marketed drugs and development candidates	-

Original paper/Published review

• Biological behavior of malignant melanoma cells correlated to their survival in vivo
  Cancer Research, 35(1):218–224, 1975