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-hMAPT: Investigating Alzheimer’s disease mechanisms.
  • 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) - -


These models are complemented by Cyagen’s Knockout Catalog
, which includes over 16,000 predeveloped knockout (KO) and conditional KO (CKO) mouse models for diseases like Parkinson’s and spinal muscular atrophy (SMA), as well as many other research fields.

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.


Click here to watch the recording now.

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.