Huntington's Disease (HD) Transgenic Model Development Service

Huntington's Disease (HD) Transgenic Models, Accelerate Your Drug Discovery Process! Are you currently facing long drug development cycles and challenges in obtaining accurate preclinical data for neurodegenerative diseases? Our HD transgenic models service helps you accelerate drug discovery and obtain high-quality, translatable preclinical data through advanced genetic engineering and comprehensive model characterization. We provide the precise tools needed to navigate the complexities of HD research.

How HD Transgenic Models Can Assist Your Project

At Creative Biolabs, our HD transgenic models service is meticulously designed to provide you with precise, actionable insights for your drug discovery projects. We offer a robust platform for target validation, compound efficacy testing, and biomarker discovery, all grounded in models that faithfully recapitulate key aspects of human HD pathology. Our capabilities ensure that your preclinical research is built on a solid foundation, de-risking your development pipeline and accelerating your path to therapeutic breakthroughs.

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Workflow

Our workflow for utilizing HD transgenic models is a streamlined, collaborative process designed for efficiency and scientific rigor, suitable for visualization as a flowchart:

• Required Starting Materials: To initiate your project, clients typically provide:
  • Target Molecule Information: Details on the specific gene, protein, or pathway you aim to modulate.
  • Compound Libraries/Therapeutic Candidates: Information on the agents you wish to test, including their mechanism of action if known.
  • Specific Research Questions: Clear objectives for the study, such as efficacy endpoints, desired phenotypic changes, or biomarker validation goals.

• Final Deliverables: Upon project completion, you will receive:
  • Detailed Study Reports: Comprehensive documentation of the entire study, including methods, results, statistical analyses, and conclusions.
  • Raw Data Sets: All collected raw data, allowing for independent verification and further analysis.
  • Comprehensive Data Analysis Summaries: Graphical representations and summaries of key findings, highlighting significant outcomes and insights.

Estimated Timeframe: The typical timeframe for this service ranges from 12 to 24 weeks, depending on the complexity of the study design, the specific HD model chosen, the number of compounds tested, and the array of endpoints required. Factors such as compound availability and specific regulatory requirements can also influence the duration.

Why Choose Us?

Choosing CBL for your HD transgenic model research means partnering with a leader in neurodegenerative disease discovery. Our two decades of specialized experience, coupled with our commitment to scientific excellence, provide distinct advantages that accelerate your research and enhance the translatability of your findings. We offer:

• Unrivaled Expertise: Our team comprises seasoned neurobiologists and pharmacologists with deep knowledge of HD pathogenesis and preclinical modeling.
• Comprehensive Model Portfolio: Access to a diverse range of validated HD transgenic models, including N-terminal fragment, full-length HTT, knock-in, and conditional models, allowing for the most relevant study design.
• Customizable and Rigorous Studies: Flexible, custom-tailored study designs with robust, gene dose-dependent, progressive, and early-onset alterations captured through a broad spectrum of validated endpoints.
• Translational Focus: Our studies are designed to generate data that is highly predictive of human clinical outcomes, bridging the gap between preclinical discovery and clinical success.
• Accelerated Discovery: Our efficient workflows and advanced capabilities help expedite your drug discovery efforts, bringing promising therapies closer to patients.

Huntington's Disease Transgenic Models

The strategic application of these transgenic models is paramount in the race to find a cure for HD. At CBL, we leverage these sophisticated platforms to drive preclinical research across multiple therapeutic modalities:

1. N-terminal Fragment Models: Capturing Early and Severe Phenotypes

These models express only a truncated, highly toxic N-terminal fragment of the human mHTT protein. They are characterized by rapid disease progression and severe phenotypes, making them invaluable for studying acute toxicity and testing interventions aimed at early, aggressive disease stages.

• R6/2 Mouse Model: The most widely studied and well-characterized HD transgenic model. R6/2 mice carry approximately 144-150 CAG repeats and exhibit a remarkably aggressive phenotype, with motor and cognitive deficits appearing as early as 5-6 weeks of age, followed by rapid weight loss and premature death (typically by 12-15 weeks). This model is particularly useful for:
  • High-throughput screening of compounds due to its rapid and robust phenotype.
  • Investigating the early molecular events triggered by mHTT toxicity.
  • Evaluating the impact of therapies on severe motor and cognitive impairments.
• N171-82Q Transgenic HD Mice: These mice express the first 171 amino acids with 82 glutamines in the polyQ domain. They also show progressive neurological phenotypes and early death, further supporting the toxicity of N-terminal mutant HTT fragments.

2. Full-Length HTT Transgenic Models: Mimicking Chronic Disease Progression

These models incorporate the full-length human HTT gene, often under the control of its endogenous promoter, providing a more physiologically relevant context for mHTT expression. They typically exhibit a slower, more progressive disease course, better mirroring the adult-onset nature of human HD.

• YAC128 Mouse Model: Developed using a Yeast Artificial Chromosome (YAC) system, these mice express full-length human mHTT with 128 CAG repeats. They show progressive motor and cognitive deficits, along with selective striatal and cortical atrophy, making them excellent for:
  • Long-term studies of disease progression.
  • Evaluating therapies designed to slow neurodegeneration over time.
  • Investigating the impact of mHTT on specific neuronal populations.
• BACHD Mouse Model: Utilizing a Bacterial Artificial Chromosome (BAC) system, BACHD mice express full-length human mHTT with 97 CAG/CAA mixed repeats. A key advantage of BACHD mice is the stability of their CAG repeat length across generations and the inclusion of loxP sites, enabling conditional mHTT expression. This model is ideal for:

Studies requiring a more stable genetic background.

Investigating cell-autonomous mHTT toxicity and cell-type specific interventions.

Longitudinal studies of subtle behavioral and neuropathological changes.

• BAC226Q Mouse Model: A more recent advancement, this model expresses full-length human HTT with approximately 226 CAG-CAA repeats. It accurately recapitulates a broad spectrum of age-dependent and progressive HD-like phenotypes, including reduced lifespan, weight loss, motor and non-motor neurological symptoms, selective brain atrophy, striatal neuronal death, mHTT aggregation, and gliosis. This model offers a comprehensive platform for:
  • Holistic assessment of therapeutic efficacy across multiple HD symptoms.
  • Detailed neuropathological studies.

Customer Reviews

"Exceptional Efficacy Data: Using CBL's Huntington's Disease Transgenic Models in our research has significantly improved our ability to generate robust efficacy data for our novel compounds. The precision of their zQ175 model allowed us to observe subtle yet critical phenotypic improvements that were previously undetectable." (March 2024), Dr. Aa Sh.

"Streamlined Preclinical Validation: CBL's expertise with the R6/2 model greatly facilitated our high-throughput screening efforts. Their detailed behavioral assessments provided clear insights, saving us considerable time and resources in identifying promising drug candidates." (January 2024), Dr. Jn De.

"Unparalleled Model Characterization: The comprehensive characterization of their BACHD models, including the stability of CAG repeats and conditional expression capabilities, was crucial for our cell-type specific toxicity studies. It allowed us to pinpoint the exact neuronal populations affected by our therapeutic intervention." (February 2024), Dr. La Mz.

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What We Can Offer

At CBL, we are committed to providing unparalleled support for your HD research. Our HD transgenic models service offers a suite of capabilities designed to accelerate your drug discovery and development process, ensuring you gain the most relevant and reliable data.

• Comprehensive Model Portfolio: Access to a diverse and rigorously validated range of HD transgenic models, including N-terminal fragment, full-length HTT, knock-in, and advanced conditional models, enabling the selection of the most appropriate platform for your specific research questions.
• Customized Study Design & Execution: Tailored study protocols and meticulous execution of in vivo experiments, ensuring precise administration of compounds, careful monitoring of animal health and behavior, and systematic data collection across all relevant endpoints.
• Advanced Phenotypic Assessment: Utilization of a broad spectrum of validated endpoints, including detailed behavioral assessments (motor, cognitive), cutting-edge neuroimaging (MRI, MRS), and comprehensive biomarker analysis (mRNA, protein, neuropathological markers), to capture robust, gene dose-dependent, progressive, and early-onset alterations.
• Expert Data Analysis & Interpretation: In-depth analysis of complex datasets by our seasoned neurobiology experts, providing clear interpretations, identifying significant trends, and offering strategic recommendations for your next steps.
• Translational Relevance: Our commitment to using models that faithfully recapitulate human HD pathology ensures that the data generated is highly predictive of clinical outcomes, enhancing the translational potential of your therapeutic candidates.
• Accelerated Research Timelines: Efficient workflows and streamlined processes, from consultation to final reporting, designed to de-risk your pipeline and significantly expedite your drug discovery journey.
• Dedicated Scientific Partnership: Collaborate with a team that possesses over 20 years of specialized experience in neurodegenerative disease research, offering unparalleled scientific guidance and support throughout your project.

Related Services

To further support your HD research, CBL offers a suite of complementary services and specialized options:

• In Vitro HD Assays: Explore our cell-based assays for high-throughput screening and target validation, providing a crucial initial step in drug discovery.
• Biomarker Development & Validation: Leverage our expertise in identifying and validating novel biomarkers for HD, essential for tracking disease progression and treatment response.
• Custom Model Generation: For highly specialized research needs, we can explore the generation of custom HD models tailored to your unique specifications.
• Neuroinflammation Studies: Investigate the role of neuroinflammation in HD pathogenesis and evaluate anti-inflammatory therapeutic strategies.

At CBL, we are dedicated to accelerating your HD research and bringing hope to patients worldwide. Our comprehensive expertise in transgenic models provides the essential tools for understanding HD and developing effective therapies. Contact our team for more information and to discuss your project.