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Báo cáo sinh học: " Human umbilical cord blood-derived mononuclear cell transplantation: case series of 30 subjects with Hereditary Ataxia'

Tuyển tập báo cáo các nghiên cứu khoa học quốc tế ngành hóa học dành cho các bạn yêu hóa học tham khảo đề tài: Human umbilical cord blood-derived mononuclear cell transplantation: case series of 30 subjects with Hereditary Ataxia | Yang et al. Journal of Translational Medicine 2011 9 65 http www.translational-medicine.eom content 9 1 65 JOURNAL OF TRANSLATIONAL MEDICINE RESEARCH Open Access Human umbilical cord blood-derived mononuclear cell transplantation case series of 30 subjects with Hereditary Ataxia 1 71 1 1 r- s z-1 3 1 1 1 Wan-Zhang Yang Yun Zhang Fang Wu Min Zhang SC Cho Chun-Zhen Li Shao-Hui Li Guo-Jian Shu V r I I V I Z i c Z Z t 1 l I I ĩ s 1 Vi I Z t 1 c II I I - Z t 2 c I I - Z t 2h bbh XAZ cd s z l I I n 1 E I r I m 4 m z l You-Xiang Sheng Ning Zhao Ying lang Shu Jiang Shan Jiang Matthew Gandjian Thomas E Ichim ana Xiang Hu2 Abstract Background The differential diagnosis for hereditary ataxia encompasses a variety of diseases characterized by both autosomal dominant and recessive inheritance. There are no curative treatments available for these neurodegenerative conditions. This open label treatment study used human umbilical cord blood-derived mononuclear cells CBMC combined with rehabilitation training as potential disease modulators. Methods 30 patients suffering from hereditary ataxia were treated with CBMCs administered systemically by intravenous infusion and intrathecally by either cervical or lumbar puncture. Primary endpoint measures were the Berg Balance Scale BBS serum markers of immunoglobulin and T-cell subsets measured at baseline and predetermined times post-treatment. Results A reduction of pathological symptoms and signs was shown following treatment. The BBS scores IgG IgA total T cells and CD3 CD4 T cells all improved significantly compared to pre-treatment values P 0.01 0.001 . There were no adverse events. Conclusion The combination of CBMC infusion and rehabilitation training may be a safe and effective treatment for ataxia which dramatically improves patients functional symptoms. These data support expanded double blind placebo-controlled studies for these treatment modalities. Background Hereditary ataxias are a heterogeneous group of neuro-degenerative