LIVE WEBINAR
Genetically Humanized Mouse Models: Bridging Human Biology and Translational Research
Conventional partial humanized mouse strains fail to replicate complete human gene regulation and splicing behavior, creating a critical translational divide between preclinical testing and human clinical outcomes. Attendees will explore validated case studies across metabolic, cardiovascular, neurological and rare disease gene therapy programs, alongside our full suite of end-to-end preclinical CRO capabilities powered by TurboKnockoutᵀᴹ editing technology.
DATE
September 11 , 2026 | 9:00 - 10:00 AM PDT
LOCATION
Online
Introduction
Genetically humanized mice are indispensable tools for disease modeling and target validation. However,
traditional models that only replace isolated coding sequences often lack the full human gene regulatory
networks and splicing contexts—creating a critical translational divide between preclinical testing and
clinical outcomes.
This session introduces fully genomically humanized mice built with the HUGO-GT™
(HUmanized Genomic
Ortholog for Gene Therapy) platform. By swapping entire mouse genes for complete human genomic
orthologs, these models successfully restore native human gene expression. Join us to explore how these
advanced models overcome traditional bottlenecks and see real-world applications across ASO, siRNA, and
gene therapy programs
Summary
This webinar is designed to address the key technical drawbacks of partial
humanization strategies and showcase the transformative advantages of complete genomic humanization for
academic and translational research. We will detail the end-to-end design workflow and explain how
preserved
human intronic, regulatory, and splicing sequences eliminate the critical gaps seen in limited
coding-region-only lines.
Attendees will explore concrete applications of these models in gene therapy and drug discovery,
highlighted through:
- Targeted Case Studies: In-depth performance data for familial hypercholesterolemia and spinal muscular atrophy (SMA).
- Therapeutic Modality Testing: Validating in vivo efficacy and clinical translatability for ASO, siRNA, and therapeutics.
- Broad Indication Applications: Exploring our library of ready-to-use HUGO-GT™ models spanning neurological, ophthalmic, metabolic, cardiovascular, and rare disease research.
- Advanced Model Generation: An overview of how TurboKnockout™ technology streamlines complex, large-fragment genomic knock-ins to create these humanized strains efficiently.
Who Should Attend
- Principal Investigators and translational medicine researchers developing human disease animal models.
- Gene therapy discovery scientists working with ASOs, siRNA, and oligonucleotide therapeutics.
- Investigators studying rare diseases, neurology, ophthalmology, metabolic, and cardiovascular disorders.
- Biotech program leaders validating novel therapeutic targets for clinical advancement.
Key Learning Objectives
- Understand the strengths, limitations, and translational impacts of different humanization strategies.
- Learn how fully genomically humanized models better recapitulate complex human gene biology (splicing and regulation).
- Explore actionable applications of HUGO-GT™ models in gene therapy research and drug development.
- Review real-world case studies demonstrating in vivo efficacy and translational impact across multiple disease areas.
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