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| 引用本文： | ����ӱ�����. ���к�ƶѪ�Ļ�������[J]. 中国实用儿科杂志, 2018, 33(12): 982-984. DOI: 10.19538/j.ek2018120613 |
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| 作者姓名： | ����ӱ����� |
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| 作者单位： | ???????????????????о?????? ??? 523128 |
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| 摘 要： |
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| 关 键 词： | ???к??? ???????? ??????????? |
Gene therapy for thalassemia |
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| LIU Hua-ying��LI Chun-fu. Gene therapy for thalassemia[J]. Chinese Journal of Practical Pediatrics, 2018, 33(12): 982-984. DOI: 10.19538/j.ek2018120613 |
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| Authors: | LIU Hua-ying��LI Chun-fu |
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| Affiliation: | South Chun Fu??Children?? Institute of Hematology??Dongguan 523128??China |
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| Abstract: | ??Gene therapy for thalassemia is based on gene-modified autologous hematopoietic stem cell transplantation. At present??preclinical and clinical studies have proved the safety and efficacy of lentivirus vector gene therapy??but there are still many factors limiting its clinical application??such as the quantity and quality of effective stem cells??the efficiency of gene transduction??level of gene expression??and toxicity of pre-transplantation preconditioning regimen. |
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| Keywords: | thalassemia gene therapy induction of multifunctional stem cells |
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