Why is the mouse model prioritized as the preferred animal model for gene therapy research?

Why are mice models prioritized in gene therapy research as the preferred animal model?

In this issue, we invited Mr. Han Lanqing, Director of the Artificial Intelligence Innovation Center of Tsinghua Pearl River Research Institute and Executive Secretary-General of the Guangzhou Rare Disease Gene Therapy Alliance. According to Mr. Han, gene therapy is different from traditional therapies, as it applies genetic engineering techniques to introduce normal genes into a patient's cells to correct defective genes and ultimately cure the disease. As gene therapy research involves gene editing, sequence replacement, and other experiments on animal embryonic stem (ES) cells, it is more suitable to use the mouse model from a test cycle and operational perspective.

Gene Therapy in Ophthalmology: New Opportunities for Vision Restoration

Genetic eye disorders are caused by genetic defects and often present as familial diseases. There are currently about 5 million blind people in China, of which genetic eye diseases account for about one-third of blindness and visual impairment in children, and 80% of blindness and severe visual impairment. Patients with monogenic inherited eye diseases account for about 4% of the total population. Compared with traditional medical methods, gene therapy as an innovative research and development field is experiencing vigorous development both domestically and internationally. Similarly, it has also brought significant breakthroughs to the diagnosis and treatment of genetic eye diseases. Whether it is the government, enterprises, or research institutions, all are actively promoting the industrialization of gene therapy drugs for ophthalmology.

To this end, the Tsinghua Pearl River Research Institute, in collaboration with Seayou Biotech's Rare Disease Data Center (RDDC), has developed a series of AI tools such as AI mutation pathogenicity prediction and AI mutation splicing prediction tools to assist in the diagnosis and research of rare diseases, and to support the development of gene therapy drugs.

Based on this, the Guangzhou Rare Disease Gene Therapy Alliance has invited experts such as Professor Jin Zhibing from Beijing Tongren Hospital of Capital Medical University, Professor Liu Chunqiao from Zhongshan Ophthalmic Center of Sun Yat-sen University, Professor Fan Guoping from Aierkan Biotech, Dr. Zhang Wei from Jiajian Medical, Director Han Lanqing from the Artificial Intelligence Innovation Center of Tsinghua Pearl River Research Institute, and Dr. Ren Sheng from the Gene Therapy Department of Seayou Biotech to participate in a salon event with the theme "Gene Therapy in Ophthalmology: New Opportunities for Vision Restoration." They aim to explore the current state and future of gene therapy for genetic eye diseases.