HUGO-GT™ Next-Generation Humanized Models

The humanized mouse model is a powerful tool that excels in replicating human physiological and pathological characteristics, outshining traditional transgenic (Tg) animal models. This makes it the preferred choice for studying human diseases and assessing the safety and effectiveness of potential therapeutics. The 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.

Despite their popularity, common humanized models — including transgenic (Tg) mice, coding sequence (CDS), and single-exon humanized mice — fall short in achieving full human gene integration into the mouse genome. These current-generation models come with notable limitations such as random insertion, complex genetic backgrounds, and inadequate humanized regions.

To advance our understanding of disease mechanisms and drug development, there is a pressing need for full-length genomic DNA humanized mice. These models can faithfully replicate human gene expression patterns, regulations, and functional properties in a mouse model. However, replacing the entire genomic DNA sequence poses technical challenges as introducing large exogenous sequences may impact the expression and regulation of endogenous genes, presenting a significant obstacle.

Full-Length Genomic Sequence Humanized HUGO Mouse Models

In response to these demands, Cyagen has introduced the HUGO-GT™ (Humanized Genomic Ortholog for Gene Therapy) program. We employ our proprietary TurboKnockout-Pro technology to perform in-situ replacement of the targeted mouse endogenous gene, creating full-length genomic sequence humanized mouse models with a broader range of intervention targets.

Our HUGO-GT™ mice utilize highly efficient large-fragment vector fusion technology, serving as a versatile template for customized targeted mutagenesis. This approach allows us to provide clinically relevant humanized mouse models closely aligned with real-world biological mechanisms.

In addition to our mouse models, we offer Contract Research Organization (CRO) services in various fields, including ophthalmology, neuroscience, tumor immunology, and other disease areas. Our aim is to empower research on genetic diseases and facilitate the development of gene therapy drugs.

Advantages of HUGO-GT™ Genomic DNA Humanized Model

1. In Situ Gene Replacement: Full genomic sequences (UTR, exon, intron) ensure accurate human gene function within the mouse genomic context and in vivo environment.
2. Minimal Gene Regulation Disruption: Maintains natural regulation and expression patterns, preserving different isoforms across tissues and cell types.
3. Flexible and Efficient: Initial wild-type humanization on mouse ES cells allows rapid introduction of disease-causing mutations, facilitating drug testing and research.
4. Superior Accuracy: Reduces artifacts seen in other models, providing phenotypes that closely resemble human conditions.
5. Comparative Superiority: Outperforms models with simple point mutations, CDS-only replacements, and BAC transgenes with random integration.