Research Progress of AAV-Mediated Gene Therapy for Hemophilia
DAI Xinyue, ZHANG Lei*
Gene therapy has great potential to produce normal levels of FVIII or FIX in patients with hemophilia. After nearly 30 years of continuous development in this field, considerable progress has been made, but there is still much room for improvement. AVV is the primary carrier of gene therapy for hemophilia. Researches will focus on improving the design of viral capsids, transgenes and promoters in order to pursue higher transduction efficiency, lower immune response and predictable treatment results. Compared with the almost 100% transduction efficiency of animal models, there are still shortcomings in achieving high transduction efficiency and high expression of coagulation factors in human hepatocytes. At the same time, attention should be paid to avoid the risk of cell stress caused by protein overload. Although gene therapy for hemophilia still faces specific challenges, safer, more effective and even personalized treatment plans will be developed in the future to truly improve the quality of life of hemophilia patients and even cure them.
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