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Disease Research Models

Therapeutic Area Mouse Models

Since 1998, ingenious targeting laboratory has generated custom mouse models supporting research across every major therapeutic area. Our 2,500+ completed projects span oncology, neuroscience, metabolic disease, immunology, cardiovascular research, rare disease, and ophthalmology, contributing to over 800 peer reviewed publications.

Whether you are studying disease mechanisms, validating therapeutic targets, or testing drug candidates, ingenious targeting laboratory provides the custom knockout, knockin, and humanized models optimized for your specific disease research needs.

2,500+
Projects Completed
800+
Publications
26+
Years Experience
100%
Success Rate

Explore Therapeutic Areas

Custom mouse models for research across major disease categories, supporting both basic research and therapeutic development programs.

Oncology Mouse Models

Cancer research requires models that accurately recapitulate tumor biology, immune interactions, and therapeutic responses. ingenious targeting laboratory supports oncology programs with tumor suppressor knockouts, oncogene models, and humanized immune checkpoint mice for immuno oncology studies.

Key Applications

  • Tumor suppressor knockout models enable study of cancer initiation and progression
  • Conditional approaches allow tissue specific or temporal control over tumor development
  • Germline knockouts model inherited cancer syndromes
  • Humanized checkpoint models including PD1, PDL1, CTLA4, LAG3, and TIM3 for immuno oncology
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Neuroscience Mouse Models

Neurological and neurodegenerative disease research demands models that reproduce disease pathology while enabling mechanistic studies. ingenious targeting laboratory has generated models for Alzheimer disease, Parkinson disease, ALS, Huntington disease, epilepsy, autism spectrum disorders, and other neurological conditions.

Key Applications

  • Alzheimer disease models incorporate mutations in APP, presenilin genes, or tau
  • Movement disorder research uses alpha synuclein models for Parkinson disease
  • SOD1 or TDP43 models for ALS and huntingtin models for Huntington disease
  • Reporter knockins enable cell population tracking in neural circuits
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Metabolic Disease Mouse Models

Metabolic research spans diabetes, obesity, fatty liver disease, and lipid metabolism disorders. ingenious targeting laboratory provides models for studying metabolic pathways, testing therapeutic interventions, and understanding disease progression.

Key Applications

  • Diabetes models address type 1 autoimmune mechanisms and type 2 insulin resistance
  • Tissue specific knockouts in liver, adipose, muscle, or pancreas reveal organ contributions
  • NASH and obesity research with models developing hepatic steatosis and fibrosis
  • Humanized models enable testing of human specific therapeutic targets
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Immunology Mouse Models

Immune system research requires models that accurately reflect human immunology while enabling mechanistic studies. ingenious targeting laboratory supports autoimmune disease research, inflammatory conditions, and basic immunology with knockout, knockin, and humanized models.

Key Applications

  • Autoimmune disease models for rheumatoid arthritis, lupus, and multiple sclerosis
  • Inflammatory bowel disease and other inflammatory conditions
  • Conditional knockouts in specific immune cell populations
  • Humanized immune models express human cytokines, receptors, or immune checkpoints
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Cardiovascular Mouse Models

Cardiovascular research encompasses atherosclerosis, heart failure, hypertension, cardiac fibrosis, and vascular disease. ingenious targeting laboratory provides models for studying cardiovascular pathophysiology and testing therapeutic approaches.

Key Applications

  • Atherosclerosis models combine lipid metabolism gene modifications with ApoE or LDLR deficiency
  • Conditional knockouts enable tissue specific studies in endothelium or smooth muscle
  • Cardiac disease models address heart failure, arrhythmia, and fibrosis
  • Ion channels, sarcomeric proteins, or signaling pathway component modifications
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Rare Disease Mouse Models

Rare and orphan diseases affect small patient populations but often have high unmet need for effective therapies. ingenious targeting laboratory supports rare disease research with models for muscular dystrophies, lysosomal storage disorders, cystic fibrosis, and other inherited conditions.

Key Applications

  • Muscular dystrophy models carry mutations in dystrophin, dysferlin, or other muscle proteins
  • Enable study of disease mechanisms and testing of gene therapy approaches
  • Lysosomal storage disorder models reproduce enzyme deficiencies
  • Platforms for enzyme replacement and gene therapy development
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Ophthalmology Mouse Models

Vision research addresses retinal degenerations, glaucoma, macular degeneration, and other ocular conditions. ingenious targeting laboratory provides models for studying photoreceptor biology, retinal pigment epithelium function, and visual pathway development.

Key Applications

  • Retinal degeneration models carry mutations in rhodopsin, peripherin, or other photoreceptor genes
  • Conditional approaches enable temporal control over degeneration onset
  • Reporter knockins enable visualization of specific retinal cell populations
  • Tracking of cell fate during development and disease
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What Researchers Say

The Hephaestin flox model ingenious has made for us has been great. It has helped generate eight research publications.

Joshua Dunaief, PhD, MD

University of Pennsylvania

Start Your Project

Ready to discuss mouse models for your therapeutic area research? Our scientific team provides complimentary project consultation to help you design the optimal model for your disease research.

✦ New for 2026

Breeding Scheme Architect

Plan complex multi-allele breeding strategies, calculate expected genotype ratios, and estimate time to experimental cohorts—all before starting your project.

Visualize multi-generation breeding paths
Calculate Mendelian ratios automatically
Estimate timeline to study-ready cohorts

Free Research Tool

No account required

Allele 1Gene-flox (conditional)
Allele 2Cre-driver (tissue-specific)
TargetHomozygous knockout

→ 3 generations to target genotype

Lab Signals

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Frequently Asked Questions

Therapeutic area models include knockout models (loss-of-function studies), knockin models (point mutations, reporters, humanized sequences), conditional models (tissue-specific or temporal control), and humanized models (for therapeutic testing). Selection depends on disease mechanism, research question, and therapeutic strategy.

Model type selection depends on research question: use knockout for loss-of-function studies, knockin for patient mutations or reporters, conditional for tissue-specific studies or essential genes, and humanized for therapeutic testing requiring human targets. Our scientific consultants help match model type to research goals.

Yes. Models can be combined across therapeutic areas for multi-disease studies, comorbidity research, or complex disease modeling. For example, metabolic disease models can be combined with cardiovascular models, or oncology models can be combined with immunology models for immuno-oncology research.

Therapeutic area models support drug development through target validation (confirming target relevance), efficacy testing (preclinical evaluation of drug candidates), biomarker discovery (identifying disease markers), and safety assessment (evaluating on-target and off-target effects). Humanized models enable testing of human-specific therapeutics.

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