HUGO-GT™ Humanized Mice for CNS Drug Discovery
Neurological diseases, such as Alzheimer’s disease, Parkinson’s disease, and epilepsy, remain some of the most challenging areas of research in biomedical science. The need for advanced and reliable animal models is more crucial than ever to uncover the pathological mechanisms behind these diseases and develop effective treatments. Cyagen’s innovative HUGO-GT™ humanized mouse models and an extensive collection of research-ready models offer comprehensive solutions to meet the growing demands of neuroscience research.
Why Use Humanized Mouse Models?
The humanized mouse model is a powerful tool that excels in replicating human physiological and pathological characteristics, outshining traditional transgenic (Tg) humanized animal models. This enhanced alignment with human biology leads to more precise predictions of drug responses and disease mechanisms, potentially streamlining the translation from preclinical research to clinical applications.
HUGO-GT™ Humanized Mouse Models: A Next-Generation Solution
Cyagen’s HUGO-GT™ (Humanized Genomic Ortholog for Gene Therapy) models set a new standard in replicating human physiological and pathological traits, surpassing traditional transgenic models. By aligning closely with human biology, these models provide more accurate predictions of drug responses and disease mechanisms, reducing the gap between preclinical and clinical research.
Commonly used humanized models, such as Tg mice and coding sequence (CDS) replacements, face challenges like random insertion, complex genetic backgrounds, and limited humanization regions. To address these issues, Cyagen employs full-length genomic DNA replacements in HUGO-GT™ models. This approach faithfully replicates human gene expression, regulation, and functionality, overcoming technical barriers associated with introducing large DNA sequences into the mouse genome.
Full-Length Genomic Sequence Humanized HUGO Mouse Models
HUGO-GT™ utilizes Cyagen’s proprietary TurboKnockout-Pro technology for in-situ replacement of mouse genes with full-length genomic sequences (exons, introns, and UTRs) of their human counterparts. This enables precise genetic modifications, supporting disease-relevant studies and therapeutic development. The platform’s large-fragment vector technology allows for customized mutagenesis, creating models closely aligned with clinical research needs.
This approach allows for highly accurate representation of human gene functions and regulatory mechanisms in animal models, making them ideal for translational neuroscience research.
Key Features of HUGO-GT™ Full Genomic Humanization Models:
- In Situ Gene Replacement: Utilizes full genomic sequences to ensure accurate replication of human gene functions.
- Minimal Gene Regulation Disruption: Maintains natural regulation patterns across different tissues.
- Disease-Relevant Mutations: Supports the rapid development of disease-causing mutations for drug testing.
- Superior Accuracy: Overcomes limitations of current-generation models, providing human-relevant phenotypes.
Neuroscience-Focused HUGO-GT™ Humanized Mouse Models:
Cyagen offers a range of HUGO-GT™ models designed specifically for neurological research, including:
- B6-hMECP2: Studying Rett syndrome and other neurodevelopmental disorders.
- B6-hTARDBP: Enabling research into amyotrophic lateral sclerosis (ALS).
These models provide unparalleled opportunities to explore the genetic underpinnings of neurological diseases and test innovative therapeutic interventions.
Expanding the Possibilities: Genetic and Induced Models
In addition to the HUGO-GT™ humanized mouse model platform, Cyagen offers an extensive collection of research-ready genetic and drug-induced rodent models, validated for a variety of neurological disorders:
Gene Edited Models:
- AD-M1: Transgenic model for research on Alzheimer's Disease (AD), Cerebral Amyloid Angiopathy (CAA), and Notch signaling pathway.
- FVB-hHTT Q150 KI: A precise model for Huntington’s disease research from pathogenesis to drug development and efficacy evaluations.
- DMD-Q995: A valuable tool for studying the mechanisms of Duchenne muscular dystrophy (DMD) and for screening potential therapeutic agents.
Induced Models:
- 6-OHDA Treated Rats: 6-Hydroxydopamine (6-OHDA) is a widely used neurotoxin for modeling the degeneration of dopaminergic (DA) neurons in Parkinson’s disease (PD) research. Commonly employed to investigate disease pathophysiology, evaluate neuroprotective strategies, and assess potential therapeutic interventions
- MPTP-treated Mice: Various dosing regimens of MPTP (1-methyl-4-phenyl-1,2,3,6-tetrahydropyridine) administered intraperitoneally have been employed to replicate different aspects of PD.
- Chronic Unpredictable Mild Stress (CUMS) Model: This model is currently the most widely used, reliable, and effective rodent model for studying depression and major depressive disorder.
Research-Ready Neurological Disease Models
Our extensive collection of neurological disease mouse models cover a wide range of targeting methods, including gene knockout, conditional knockout, point mutation, transgenic, and humanized models.
Explore Our Research-Ready Neurological Disease Models:
| Product Number | Product | Strain Background | Application |
| C001427 | B6-hSNCA | C57BL/6NCya | Parkinson's disease |
| C001504 | B6-hSMN2(SMA) | C57BL/6NCya | Spinal muscular atrophy (SMA) |
| C001518 | DMD-Q995* | C57BL/6JCya | Duchenne muscular dystrophy (DMD) |
| C001410 | B6-htau | C57BL/6JCya | Frontotemporal dementia, Alzheimer's disease, and other neurodegenerative diseases |
| C001437 | B6-hIGHMBP2 | C57BL/6NCya | Spinal muscular atrophy with respiratory distress type 1 and Charcot-Marie-Tooth disease type 2S |
| C001418 | B6-hTARDBP | C57BL/6JCya | Amyotrophic lateral sclerosis, frontotemporal dementia, and other neurodegenerative diseases |
| C001398 | B6-hATXN3 | C57BL/6NCya | Spinocerebellar ataxia type 3 |
| C001568 | B6-hMECP2 | C57BL/6NCya | Rett syndrome |
| C001569 | B6-hMECP2*T158M | C57BL/6NCya | Rett syndrome |
| I001124 | B6-hLMNA | C57BL/6NCya | Progeria syndrome |
| CG0015 | 6-OHDA Treated Rats | - | Parkinson's disease (PD) |
| CG0016 | CUMS Model | C57BL/6JCya | Depression |
| C001210 | AD-M1 | C57BL/6JCya | Research on Alzheimer's Disease (AD), Cerebral Amyloid Angiopathy (CAA) and Notch signaling pathway. |
| C001541 | AD-M2 | C57BL/6JCya | Research on Alzheimer's Disease (AD), Cerebral Amyloid Angiopathy (CAA), Notch signaling pathway and other neurodegenerative diseases. |
| I001019 | FVB-hHTT Q150 KI | FVB/NJCya | Development and screening of therapeutic drugs for Huntington's disease; Evaluation of therapeutic drug efficacy and safety for Huntington's disease; Research on the pathogenesis of Huntington's disease. |
| - | MPTP-treated Mice | - | Parkinson's disease (PD) |
| - | Chronic Compression Injury Model of the Sciatic Nerve (CCI) | - | - |
Neuroscience CRO Platform: Comprehensive Solutions for Research
Cyagen’s Contract Research Organization (CRO) platform provides a one-stop solution for neuroscience studies. Services include model development, breeding, drug administration, behavioral testing, and phenotype assessments. With state-of-the-art neurobehavioral analysis platforms, Cyagen ensures high-quality results to accelerate your research.
Watch the Neuroscience HUGO-GT™ Webinar
To learn more about how HUGO-GT™ models are transforming neuroscience research, watch Cyagen’s exclusive webinar recording. The session covers:
- An introduction to HUGO-GT™ models and their applications.
- A case study on the hMECP2 model for Rett syndrome research.
- Insights into Cyagen’s CRO platform for neuroscience studies.
Why Choose Cyagen for Your Research Needs?
With over 78,400 models developed and 10,925 publications citing its services, Cyagen has established itself as a trusted partner for researchers worldwide. By combining innovative humanized models, validated genetic and induced models, and comprehensive CRO services, Cyagen offers the tools and expertise to advance your neuroscience research.
Complimentary Consultations
Cyagen’s team is available for free consultations to discuss custom model creation and tailored solutions for your specific research goals. Get a project quote from our custom model generation experts.
Start accelerating your research with Cyagen’s cutting-edge solutions and models for neurological disease studies—Contact Us today to explore how we can support your projects.
