Clinical and Molecular Mechanism Research on β- Thalassemia Treated with "Yi Sui Sheng Xue Ling"
 
　　
Objective: To observe the clinical effects of β- Thalassemia treated with Yi Sui Sheng Xue Ling and analyzes the molecular biological basis of the therapeutic effects.

Methods: According to the TCM theory, the kidney produces marrow. We used the composite recipe of nourishing kidney and supplementing blood (NK&SB) to treat β –Thalassemia patients for a 3-month treating course in Guangxi Province, the high incidence area of the disease. Then we rechecked and visited them for 3-6 months after the treatment. Through analyzing genes of the patients systematically with PAGE, PCR, PCR-SSCP, RT-PCR, DNA sequences analysis, MRNA gene expression technique etc, we studied the molecular mechanism of the effect from the relationship between genes mutation and the effect to genes expression and regulation. Clinical data prove that the NK&SB has better effect in treating β-Thalassemia compared with the western herb Myleran, which also shows the advantages of TCM. Data of genes analysis prove that TCM treatment does not change genetic mutation, but it can obviously increase Hb, HbF, the ratio of γ to β + γ of Globulin chains, mRNA expression of β -Globin and GM-CSF MRNA expression in the blood of patients. The further study testifies that NK&SB can obviously increase GM-CFV, BFU-E and CFU-E in the marrow of the mice. 

Results: NK&SB have obvious effect on β -Thalassemia. The research has three characteristics as follows:
(l) It brings forward the hypothesis that kidney produces marrow is the TCM Theoretical core of treating β -Thalassemia and testifies its objectivity;
(2) It suggests the possible mechanism of treating β -Thalassemia with TCM that is TCM can strengthen vigor, of the organism, unlock γ -gene, stimulate mRNA expression of Globin and induce increasing of synthesis of HbF to compensate the defect of β -gene and achieve the goal of treatment.
(3) The study explains the molecular mechanism of TCM theory that kidney produces marrow at genetic and molecular level. It also facilitates the study of the classical theory to reach the international leading role.

Conclusion: The study finds a new path to treat β -Thalassemia with TCM and provides scientific basis for treating the disease both in theory and practice.

By Wu Zhikui, Huang Youwen, etc.