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| Clinical efficacy and molecular mechanism of nourishing shen and supplementing marrow principle in treating β-thalassemia |
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| KeyWord:nourishing Shen and supplementing marrow principle Shen producing marrow β-thalassemia gene analysis mRNA gene expression molecular mechanism |
| Author Name | Affiliation | E-mail | | Wu Zhi-kui | Department of Molecular-Biology, Guang’anmen Hospital, China Academy of Traditional Chinese Medicine, 100053, Beijing | zhikuiwu@yahoo.com | | Fang Su-ping | Department of Molecular-Biology, Guang’anmen Hospital, China Academy of Traditional Chinese Medicine, 100053, Beijing | | | Zhang Xin-hua | Department of Molecular-Biology, Guang’anmen Hospital, China Academy of Traditional Chinese Medicine, 100053, Beijing | | | Cai Hui-guo | Institute of Hematology, Chinese Academy of Medical Sciences, China | | | Wang Lei | Department of Molecular-Biology, Guang’anmen Hospital, China Academy of Traditional Chinese Medicine, 100053, Beijing | | | Yi Jie | Department of Molecular-Biology, Guang’anmen Hospital, China Academy of Traditional Chinese Medicine, 100053, Beijing | | | Chai Li-min | Department of Molecular-Biology, Guang’anmen Hospital, China Academy of Traditional Chinese Medicine, 100053, Beijing | | | Lu Xin-xia | Department of Molecular-Biology, Guang’anmen Hospital, China Academy of Traditional Chinese Medicine, 100053, Beijing | | | Chen Yu-ying | Department of Molecular-Biology, Guang’anmen Hospital, China Academy of Traditional Chinese Medicine, 100053, Beijing | | | Huang You-wen | The 303 Hospital of PLA, China | | | Wang Rong-xin | The 303 Hospital of PLA, China | | | Chen Pei-zhen | Institute of Hematology, Chinese Academy of Medical Sciences, China | |
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| Abstract: |
| Objective: To explore the possibility of using traditional Chinese medicine (TCM) in treating β-thalassemia, its clinical effect and molecular mechanism of the action.Methods: According to the TCM theory of “Shen producing marrow”, the composite recipe, Yisui Shenxueling Granule (YSSXL), consisting of Chinese drugs for nourishing Shen and supplementing marrow (NS & SM) was given orally to 78 patients with β-thalassemia (49 of the severe type and 29 of moderate type), 3 times a day, 10 g each time (for children, the dose would be reduced properly), with 3 months as one therapeutic course, and no blood transfusion used in the course. The clinical therapeutic efficacy and hematologic parameters in patients were observed, and systemic gene analysis was conducted with PAGE, PCR, PCR-SSCP, RT-PCR and DNA sequences analysis and mRNA detection, in order to study the molecular mechanism from the relationships between genetic mutation and clinical efficacy, gene expression and its regulation.Results: YSSXL showed obvious therapeutic effect in treating β-thalassemia. Gene analysis revealed that it did not change the genetic mutation type, but could obviously increase hemoglobin, fetal hemoglobin (HbF), γ/(β+γ) globin ratio, γ-globin mRNA expression and GM-CSF mRNA expression in patients, as well as the GM-CSFmRMA in marrow of mice after60Co radiation.Conclusion: YSSXL has a remarkable therapeutic effect on β-thalassemia, and its possible mechanism is its action in unlocking γ-gene, increasing the γ-globin expression and enhancing HbF synthesis so as to compensate for the gene defect. This study has opened a new path for the treatment of β-thalassemia with TCM. This item was supported by National Funds of Natural Sciences (No. 30171199) and Natural Science Foundation of Guangxi Autonomous Region (No. 014402C) |
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