| Description |
As an important breakthrough in the new medical revolution, regenerative medicine with stem cell therapy as the core has become another way of disease treatment after drug therapy and surgical treatment. However, there are still huge risks associated with stem cell therapy, and a systematic scientific evaluation of the safety and efficacy of stem cell therapy is imperative. Obtaining stable animal models that closely resemble human diseases and high-quality stem cell resources are the key to a more objective evaluation of the safety and efficacy of cell therapy. Primates is considered to be a variety of the human nervous system diseases or only the most appropriate experimental animal models, therefore, the establishment and the human nervous system diseases are highly similar primate model, built from pluripotent stem cells, differentiation, encourage to full cell transplantation treatment course of preclinical safety and efficacy evaluation, It is of great significance to the establishment of clinical treatment and standard for stem cells. This project is based on the international leading primate targeted gene editing and stem cell technology platform to establish animal models of PD, ALS and RTT diseases, ESCs, iPSCs and NTESCs different types of pluristem cell lines and corresponding neural differentiation and culture systems, through different types of diseases (neurodegenerative diseases and early nervous system development abnormalities), To establish stem cell therapy strategies (transplantation cell type, differentiation system, cell repair and transplantation methods, etc.) corresponding to different disease populations (adults or minors) and different genetic mutation types of diseases, and to systematically answer the most critical questions of clinical stem cell therapy: Safety and effectiveness, and ultimately provide a theoretical and practical basis for the treatment of human neurological diseases. |
