Huntington's Disease (HD) Knock-in Model Development Service

Huntington's Disease (HD) Knock-in Models, Accelerate Your Neurodegenerative Drug Discovery! Are you currently facing challenges in accurately modeling complex genetic disorders, predicting therapeutic efficacy, or understanding early disease pathogenesis? Our Huntington's disease knock-in models help you accelerate drug discovery, obtain high-quality preclinical data, and gain deeper insights into HD progression through genetically precise reproductions of the human condition and comprehensive phenotypic characterization.

How Our Huntington's Disease Knock-in Models Can Assist Your Project

Creative Biolabs' HD knock-in models provide a robust platform to tackle critical challenges in neurodegenerative drug development. We deliver genetically precise models that faithfully recapitulate human disease pathology, enabling accurate target validation, efficacy testing, and a deeper understanding of disease mechanisms. This allows you to identify effective therapeutic candidates with higher confidence, streamline your preclinical pipeline, and reduce the risk of clinical trial failures.

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Workflow:

Our workflow for providing HD knock-in models services is designed for clarity and efficiency, ensuring a seamless collaborative experience.

• Required Starting Materials: To initiate a project, clients typically provide detailed project objectives, specific research questions related to HD pathogenesis or therapeutic intervention, and, if applicable, initial data on compounds or gene targets they wish to test. We also require information on desired CAG repeat length for the knock-in model, and any specific genetic background preferences.

• Final Deliverables: Upon completion, clients receive detailed study reports, comprehensive raw data sets, statistical analyses of all collected parameters, and a scientific interpretation of the findings. These may include behavioral score sheets, histopathological images, quantitative PCR data for gene expression, and Western blot data for protein analysis.
• Estimated Timeframe: The typical timeframe for HD knock-in models projects ranges from 12 to 36 weeks, depending on the complexity of the study design, the specific knock-in line selected, the number of experimental cohorts, and the depth of ex vivo analysis required. Longer longitudinal studies will naturally extend the duration.

Why Choose Us?

CBL offers unparalleled expertise and a proven track record in developing and utilizing HD knock-in models, positioning us as your ideal partner in neurodegenerative research. Our commitment to scientific excellence ensures that your projects yield robust, reliable, and clinically relevant data.

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Huntington's Disease Knock-in Models

HD knock-in models are advanced research tools that accurately replicate human HD by introducing the expanded CAG repeat into the endogenous mouse Htt locus. This ensures mutant huntingtin is expressed physiologically under its native promoter in its full-length form, crucial for studying disease onset, progression, and therapeutic efficacy.

Among these, the zQ175 knock-in mouse, derived from a spontaneous expansion in the CAG 140 colony, carries significantly higher CAG repeat lengths (around 188 repeats) and offers a more robust and earlier phenotype compared to existing models, making it highly valuable for evaluating therapeutic approaches and early pathogenic events.

Fig.1 Loss of body weight in both heterozygous and homozygous zQ175 KI mice.¹

Customer Reviews

"Unmatched Disease Relevance: Using CBL's HD knock-in models in our research has significantly improved the preclinical validation of our novel therapeutic agents. The physiological expression and accurate disease progression, mirroring human HD, provided data that was far more predictive than previous transgenic models, allowing us to confidently move forward with lead candidates." Dr. S. N*xon, 2024.

"Comprehensive Phenotyping: The detailed behavioral, molecular, and neuropathological assessments offered by CBL's knock-in models have been invaluable. We were particularly impressed by the early detection of subtle behavioral abnormalities and the thorough analysis of mutant huntingtin aggregation, which provided critical biomarkers for our compound's efficacy. It truly allowed for a holistic understanding of our treatment's impact." Dr. A. M*ller, 2025.

"Exceptional Support and Collaboration: CBL's team provided outstanding scientific guidance throughout our project. Their deep understanding of HD pathology and the nuances of knock-in models ensured optimal experimental design and insightful data interpretation. This partnership was crucial in navigating complex research questions and accelerating our discovery efforts." Dr. J. D*vis, 2024.

What We Can Offer

At CBL, we combine our extensive expertise in neurobiology and genetic engineering with a client-focused approach to deliver superior HD knock-in models services. Our commitment is to provide you with the most accurate and predictive preclinical tools, tailored to your unique research objectives.

• Customized Model Generation & Selection: We offer a versatile range of established HD knock-in lines with varying CAG repeat lengths, allowing for the precise selection of a model that aligns with your specific disease progression and severity requirements. Beyond our existing catalog, CBL specializes in generating bespoke knock-in models to address novel research questions or incorporate specific genetic modifications, ensuring unparalleled relevance for your study.
• Comprehensive Phenotypic Characterization Capabilities: Our in-depth phenotyping services cover the full spectrum of HD pathology, including precise behavioral assessments (motor, cognitive, psychiatric), detailed neuropathological evaluations (e.g., mutant huntingtin aggregation, gliosis, axonal degeneration), and advanced molecular and biochemical analyses (e.g., transcriptional dysregulation, proteolytic cleavage, BDNF levels). This provides a holistic view of disease progression and therapeutic efficacy, empowering data-driven decisions.
• Physiological Accuracy and Predictivity: CBL's knock-in models ensure the expression of mutant huntingtin under its endogenous promoter in the full-length protein context. This physiological fidelity means that observed phenotypes and therapeutic responses are highly representative of the human condition, significantly enhancing the translatability of your preclinical data and reducing risk in clinical trials.
• Robust Quality Control and Reproducibility: We maintain stringent genetic and health quality control protocols for all our animal colonies, guaranteeing the stability and reproducibility of knock-in lines across experiments. This commitment to quality ensures consistent and reliable results, which are critical for robust drug development pipelines.
• Expert Scientific Consultation and Support: Our team of seasoned biology experts with over 20 years of experience provides unparalleled scientific consultation at every stage of your project. From experimental design and protocol optimization to data interpretation and strategic insights, we partner with you to maximize the scientific value and impact of your HD research.

Related Services

To further enhance your HD research, CBL offers a suite of complementary services designed to provide a comprehensive solution for drug discovery and development.

• In Vitro HD Assays: Explore our range of cell-based assays for initial compound screening and mechanistic studies, offering high-throughput capabilities for early-stage lead identification.
• Custom Genetic Modeling: Beyond knock-in models, we offer bespoke genetic engineering services to create highly specialized animal models tailored to unique research questions or specific genetic backgrounds.
• Pharmacokinetic/Pharmacodynamic (PK/PD) Studies: Integrate PK/PD assessments with your efficacy studies to understand drug exposure, distribution, and target engagement, crucial for optimizing dosing strategies.

Ready to accelerate your Huntington's Disease research? Contact our team for more information and to discuss your project.

Reference

1. Menalled, Liliana B., et al. "Comprehensive behavioral and molecular characterization of a new knock-in mouse model of Huntington's disease: zQ175." PloS one 7.12 (2012): e49838. Distributed under Open Access license CC BY 4.0, without modification.