On-Demand Webinar: 11:00AM - 1:00PM PT

Genetically Humanized Mouse Models: Bridging Human Biology and Translational Research

PRESENTER: 

Marvin Ouyang, MD, Ph.D. | Principal Scientist

PRESENTER'S BIO: 

As the Principal Scientist at Cyagen, Dr. Ouyang oversees the company’s scientific operations and strategic development of genetically engineered mouse model services. With over two decades of experience in the field, Dr. Ouyang has led innovations that have advanced Cyagen’s global leadership in custom model generation. He is passionate about bridging cutting-edge technologies with translational research to accelerate biomedical discoveries. Before joining Cyagen in 2012, Dr. Ouyang served as the head of Molecular Biology at Taconic Biosciences.

ABSTRACT:

Genetically humanized mouse models have become indispensable tools for understanding human disease mechanisms, evaluating therapeutic targets, and advancing translational research. Traditional humanization strategies often focus on replacing specific coding regions; however, these approaches may not fully capture the complexity of human gene regulation, splicing, and non-coding therapeutic targets.In this webinar, we will discuss the design, generation, and application of genetically humanized mouse models, with a special focus on fully genomically humanized models that replace entire mouse genes with their human genomic counterparts, including exons, introns, and regulatory elements. These advanced models preserve human gene architecture and expression patterns, enabling more clinically relevant studies of disease biology and therapeutic intervention.We will present Cyagen’s HUGO-GT® (HUmanized Genomic Ortholog for Gene Therapy) platform and demonstrate how fully genomically humanized models can accelerate the development of gene therapies, including antisense oligonucleotides (ASOs), siRNA, CRISPR-based therapeutics, and other emerging modalities. Case studies in familial hypercholesterolemia and spinal muscular atrophy will illustrate the value of these models in target validation, efficacy evaluation, and translational research.Attendees will also gain an overview of Cyagen’s growing portfolio of HUGO-GT® models across neurological, ophthalmic, metabolic, cardiovascular, and rare diseases, as well as the enabling TurboKnockout® technology that supports efficient large-fragment genomic humanization.This webinar will be of interest to researchers working in genetics, neuroscience, rare diseases, gene therapy, drug discovery, and translational medicine who are seeking more predictive preclinical models to bridge the gap between laboratory research and clinical development.Key Learning Objectives:Understand the strengths and limitations of different humanization strategies.Learn how fully genomically humanized models better recapitulate human gene biology.Explore applications of HUGO-GT® models in gene therapy research and drug development.Review real-world case studies demonstrating translational impact across multiple disease areas.