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!