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On-Demand Webinar

The Use of Animal Models in Rare Disease Therapeutic Research

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Due to their rarity and high level of symptom complexity, rare diseases are often hard to diagnose, and there is still no cure for most rare diseases and few treatments. Since 72% of rare diseases are caused by genetic disorders, gene therapy is a promising treatment option that brings hope to rare disease patients. However, significant preclinical research is needed to lay the foundation for any treatment before applying it to human beings. Genetically engineered mouse or rat models that can display some (or all) the pathological processes in an actual human patient with these diseases play an important role in rare disease research.

To understand disease mechanisms and explore effective therapeutic targets, the selection of experimental models is essential. In this on-demand Webinar, Dr. Marvin Ouyang, Executive Vice President and Chief Scientific Officer of Cyagen, explains the current research progress on rare diseases, introduces successful cases of using animal models in rare disease therapeutic research and effective gene-editing strategies that can accelerate your research progress!


In this Webinar, you will learn:
1.Introduction of Rare Diseases
2.Gene Therapy and Animal Models
3.Gene Therapy Case Study:
Case Study #1: Developmental and Epileptic Encephalopathies (DEEs)
Case Study #2: Familial Hypercholesterolemia (FH)
4.Animal Model Generation and AAV Services
ES Cell Gene Targeting
CRISPR/Cas9 System
AAV Packaging services
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About the Presenter
Dr. Marvin Ouyang
Executive Vice President and Chief Scientific Officer
Dr. Marvin Ouyang has been dedicated to the development of genetically engineered rodent models for the past 20 years, developing hundreds of transgenic and KO/KI mouse/rat models for biomedical research and drug development. Previously, he served as the senior scientist of Oklahoma Medical Research Foundation (OMRF), a scientist for US-based Thios Pharmaceuticals, and worked for Taconic Biosciences as the leader of the Molecular Biology Department. So far, he has published many papers in high-impact academic journals such as PNAS and JBC. Technical services developed by Dr. Ouyang have been directly cited hundreds of times by top international journals, including Nature.
About Cyagen

Cyagen is a 700-employee company headquartered in Santa Clara, California, with additional locations in Japan and production facilities in China. Cyagen is a leading provider of complete research model solutions. We offer a “one-stop-shop” tailored to biomedical scientists’ gene research needs.

Our comprehensive services cover the entire process of animal model generation: from transgenic/gene targeting strategy design to animal model development, breeding, cryopreservation, and animal phenotype analysis. In addition, our virus products and services are widely used in the construction of various cell models, in vivo injection of living animals, gene therapy, and other research fields.


Research Services Details
Cyagen Knockout Catalog Models Over 16,000 ready-to-use knockout (KO)/conditional knockout (cKO) mouse models
Custom Mouse & Rat Models
(Genetically Modified)
● Knockout, Conditional Knockout
● Knockin, Conditional Knockin
● Humanization
● Point Mutation
● Transgenic
Drug Screening and Assessment Mouse Models ● Genetically modified tumor mouse/rat models
● Immunodeficient mice
● Syngeneic model
● Human tumor cell line xenotransplantation (CDX) model
● Mice with reconstituted human immune system
● Immune checkpoint (ICP) humanized mice
● Models for metabolic disease and drug efficacy evaluations
Animal Model Phenotype Analysis & Supporting Services ● Animal Surgical & Disease Model Services
● Phenotype Analysis of Mouse and Rat Models
● Breeding Service
● Cryopreservation Service
Cell Products and Services ● Cell Line Generation - KO/KI/Point Mutation/Overexpression
● Viral Vector Construction
● Lentivirus Packaging
● Stem Cells
● Cell Media
● Cell Supplements

>> For more information about gene editing strategy and model selection, Contact us with your project information, and our experts will be in touch within 48 hours.