Challenges and Future Perspectives in Viral Vector Gene Therapy

Gene therapy, particularly AAV-mediated gene therapy, represents a promising new therapeutic modality that offers patients treatment possibilities beyond the reach of conventional drugs. Its well-defined therapeutic targets, clear mechanism-of-action, and the unique advantage of “one-time treatment with long-term efficacy” make gene therapy especially compelling. After half a century of research and development, the field has not only made qualitative leaps in clinical safety and efficacy but also achieved milestone breakthroughs in commercialization. China’s gene therapy field has entered a period of rapid growth, driven by global scientific progress, strong market potential, robust capital investment, and supportive policies. However, challenges remain in the development and commercialization of gene therapy products. In response to this situation, this paper systematically analyzes the core issues faced by AAV gene therapy in scientific mechanisms, clinical translation, and commercial application, proposes corresponding strategies and future development directions, and finally offers recommendations for the sustainable development of the gene therapy field in China.

CSTR: 32200.14.cjcb.2026.09.0009