Gene therapy has shown significant potential in treating various diseases,particularly inherited blood disorders such as hemophilia,sickle cell disease,and thalassemia.Advances in understanding the regulatory network ...Gene therapy has shown significant potential in treating various diseases,particularly inherited blood disorders such as hemophilia,sickle cell disease,and thalassemia.Advances in understanding the regulatory network of disease-associated genes have led to the identification of additional therapeutic targets for treatment,especially for β-hemoglobinopathies.Erythroid regulatory factor BCL11A offers the most promising therapeutic target for β-hemoglobinopathies,and reduction of its expression using the commercialized gene therapy product Casgevy has been approved for use in the UK and USA in 2023.Notably,the emergence of innovative gene editing technologies has further broadened the gene therapy landscape,presenting possibilities for treatment.Intensive studies indicate that base editing and prime editing,built upon CRISPR technology,enable precise single-base modification in hematopoietic stem cells for addressing inherited blood disorders ex vivo and in vivo.In this review,we present an overview of the current landscape of gene therapies,focusing on clinical research and gene therapy products for inherited blood disorders,evaluation of potential gene targets,and the gene editing tools employed in current gene therapy practices,which provides an insight for the establishment of safer and more effective gene therapy methods for a wider range of diseases in the future.展开更多
AIM： TO observe the therapeutic effects of new traditional Chinese medicine （TClVl） therapy on coagulation disorder and accompanying intractable jaundice in HBV-related liver cirrhosis patients. METHODS： Using str...AIM： TO observe the therapeutic effects of new traditional Chinese medicine （TClVl） therapy on coagulation disorder and accompanying intractable jaundice in HBV-related liver cirrhosis patients. METHODS： Using stratified random sampling according to fibrinogen （Fib） levels, 145 liver cirrhosis patients due to hepatitis B complicated by coagulation disorder were treated. Of them, 70 in research group were treated with TCM by ＂nourishing yin, cooling blood and invigorating blood circulation＂ and Western medicine, 75 in control group were treated with conventional Western medicine. The indexes of liver function, coagulation function and bleeding events were observed and compared. RESULTS： The prothrombin time （PT） was shorter and the fibrinogen （Fib） level was higher in the research group than in the control group （Fib = 1.6-2.0 g/L, 1.1-1.5 g/L, and ≤ 1.0 g/L）. The total bilirubin （TBIL） level was significantly lower in the research group than in the control group, except for the subgroup of FIB ≤ 1.0 g/L. CONCLUSION： TCM therapy can improve coagulation fuction and decrease TBIL.展开更多
基金supported by Research Fund of Sichuan Academy of Medical Sciences and Sichuan Provincial People’s Hospital(30420230006)the National Natural Science Foundation of China(Nos.82300142)Sichuan Science and Technology Program(2022ZYD0131)。
文摘Gene therapy has shown significant potential in treating various diseases,particularly inherited blood disorders such as hemophilia,sickle cell disease,and thalassemia.Advances in understanding the regulatory network of disease-associated genes have led to the identification of additional therapeutic targets for treatment,especially for β-hemoglobinopathies.Erythroid regulatory factor BCL11A offers the most promising therapeutic target for β-hemoglobinopathies,and reduction of its expression using the commercialized gene therapy product Casgevy has been approved for use in the UK and USA in 2023.Notably,the emergence of innovative gene editing technologies has further broadened the gene therapy landscape,presenting possibilities for treatment.Intensive studies indicate that base editing and prime editing,built upon CRISPR technology,enable precise single-base modification in hematopoietic stem cells for addressing inherited blood disorders ex vivo and in vivo.In this review,we present an overview of the current landscape of gene therapies,focusing on clinical research and gene therapy products for inherited blood disorders,evaluation of potential gene targets,and the gene editing tools employed in current gene therapy practices,which provides an insight for the establishment of safer and more effective gene therapy methods for a wider range of diseases in the future.
基金Science and Technology Agency of Guangdong Province,NO.2008B030301041
文摘AIM： TO observe the therapeutic effects of new traditional Chinese medicine （TClVl） therapy on coagulation disorder and accompanying intractable jaundice in HBV-related liver cirrhosis patients. METHODS： Using stratified random sampling according to fibrinogen （Fib） levels, 145 liver cirrhosis patients due to hepatitis B complicated by coagulation disorder were treated. Of them, 70 in research group were treated with TCM by ＂nourishing yin, cooling blood and invigorating blood circulation＂ and Western medicine, 75 in control group were treated with conventional Western medicine. The indexes of liver function, coagulation function and bleeding events were observed and compared. RESULTS： The prothrombin time （PT） was shorter and the fibrinogen （Fib） level was higher in the research group than in the control group （Fib = 1.6-2.0 g/L, 1.1-1.5 g/L, and ≤ 1.0 g/L）. The total bilirubin （TBIL） level was significantly lower in the research group than in the control group, except for the subgroup of FIB ≤ 1.0 g/L. CONCLUSION： TCM therapy can improve coagulation fuction and decrease TBIL.
