Comprehensive Huntington's Disease CRO Solutions for Drug Discovery
Advance the development of targeted therapeutics for Huntington's Disease with Cyagen's specialized research platform. We empower scientists worldwide with validated knock-in models, end-to-end CRO services, and expert strategy to accelerate the discovery of novel HD treatments.
An Integrated Approach to HD Research & Its Challenges
As a fatal genetic disorder characterized by progressive motor, cognitive, and psychiatric symptoms, HD carries a heavy burden with no available cure. Researchers face key bottlenecks, including the need for models that accurately reflect the progressive nature of the disease and the challenge of establishing robust, longitudinal assessments of therapeutic efficacy.
To overcome these hurdles, Cyagen has developed a specialized and integrated research platform. Our portfolio of knock-in models that closely mimic the human genetic condition, combined with the comprehensive in vivo and in vitro services detailed below, provides the critical tools scientists need to establish robust assessments and accelerate their HD discovery programs.
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Our comprehensive portfolio of Huntington's Disease (HD) models is centered on knock-in (KI)
technology to closely mimic the human genetic condition. The table below includes both our
validated, ready-to-use models for immediate deployment, and our advanced R&D platform for key
disease modifiers, available for custom projects and collaborative research.
| Target Class | Model Name | Key Features | Status | Details |
|---|---|---|---|---|
| HTT Pathology | FVB-hHTT Q150 KI | Knock-in of mutant human HTT exon 1 with ~150 CAG repeats | Ready-to-Use | View |
| FVB-HTT KI(nQ) | Knock-in of mutant human HTT exon 1 with ~100 CAG repeats and human HTT promoter | Ready-to-Use | View | |
| Disease Modifier | MSH3 | Humanized MSH3 (DNA Mismatch Repair) | R&D | - |
Can't find the model you need?
Explore hundreds of additional off-the-shelf neuroscience models in our MouseAtlas database, or partner with
our scientific team to develop a custom-engineered model tailored to your exact research
specifications.
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Precision Drug Administration
Our team is highly experienced in administration methodologies critical for HD
research, including systemic and direct CNS delivery routes for novel therapeutics like ASOs
and small molecules.
- Systemic Delivery: Intravenous (tail vein), Intraperitoneal, Subcutaneous, Oral Gavage, etc.
- CNS-Targeted Delivery: Stereotactic brain injection (e.g., into the striatum), Intrathecal injection, Intraventricular injection.
Neurobehavioral Assessment for HD
We provide a focused suite of behavioral paradigms essential for evaluating the core motor,
cognitive, and psychiatric-like symptoms associated with Huntington's Disease.
| Behavioral Domain | Recommended Tests for HD Research | Function Tested |
|---|---|---|
| Motor Function & Coordination | Rotarod Test | Motor coordination, balance, and motor learning deficits |
| Grip Strength | Muscle strength and progressive motor decline | |
| Balance Beam | Fine motor coordination and balance | |
| Spontaneous Activity & Anxiety | Open Field Test | Locomotor activity (hyper/hypo-activity), anxiety-like behavior |
| Cognitive Function | Y-Maze | Spatial working memory |
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We offer a specialized suite of analytical services to quantify the core
pathological hallmarks of Huntington's Disease and to elucidate the molecular mechanisms of
your therapeutic candidates.
| Analysis Category | Specific Services | Key Applications & Targets in HD Research |
|---|---|---|
| Pathology & Biomarker Analysis | Histology & IHC/IF | Visualize and quantify mutant Huntingtin (mHTT) aggregates (using antibodies like EM48) and assess neuronal loss, particularly in the striatum and cortex. |
| ELISA & Immunoassays | Accurately quantify levels of soluble and insoluble mutant Huntingtin (mHTT) in brain tissue, CSF, and other biological samples to measure target engagement. | |
| Gene & Protein Expression | Western Blot | Assess the expression levels of total and mutant Huntingtin (HTT) protein, a critical readout for evaluating the efficacy of HTT-lowering therapeutics (e.g., ASOs, siRNAs). |
| RT-PCR & qPCR | Analyze the expression of the HTT gene to confirm target engagement and investigate downstream transcriptional changes. |
Case Studies
Case Study 1: FVB-hHTT Q150 KI: Profiling Progressive HD-like Deficits
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Case Study 2: FVB-HTT KI(nQ): Validating the nQ Control Model
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Why Partner with Cyagen?
Clinically Relevant Models
Gain access to validated, knock-in models that exhibit progressive, age-dependent phenotypes highly relevant to the human condition.
Integrated Efficacy Platform
Utilize our one-stop services—from longitudinal behavioral monitoring to terminal pathological and biochemical analysis.
Data-Driven Decision Support
Our stringent quality control and commitment to reproducibility deliver reliable data for your project decisions.
Expert-Led Study Design
Collaborate with our senior scientists for end-to-end support, from model selection to customized protocol design.
Request a Preclinical CRO Services Consultation
Partner with Cyagen to advance your preclinical studies. Share your project goals with us and receive customized support.
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