基因治疗与心力衰竭

Gene therapy and heart failure

目的:探讨基因治疗方案的有效性和安全性。资料来源:应用计算机检索PUBMED1980-01/2006-10和EMCC 1994-10/2006-10与心力衰竭的基因治疗相关文章,检索词“Gen Therapy,Heart Failure”,并限定文章语言种类为“English”;同时计算机检索CMCC 1994-01/2006-10的相关文章,限定文章语言种类为中文,检索词“基因治疗、心力衰竭”。资料选择:对资料进行初审,选取试验包括上述治疗组和对照组的文献,然后筛除明显不随机临床试验的研究,对剩余的文献开始查找全文,进一步判断是否为RCT。纳入标准为:①试验包含平行对照组,即一般药物治疗。②治疗组为转基因治疗。资料提炼:共收集到49篇相关的随机和未随机试验,31个试验符合纳入标准。其余的排除。资料综合:31个试验包括478例动物模型,分别对应用不同靶点的转基因或基因敲除等方案进行治疗者予以评价。31篇文章中有10篇未达到预期的治疗目标,主要是在目的基因的选择及转染载体的选择方面出现了偏差。结论:虽动物试验取得了一定的成果,但尚无人体基因治疗的文献报道,随着基因技术的进步,基因治疗有望成为治疗心力衰竭的新途径。

OBJECTIVE： To investigate the validity and safety of gene therapy. DATA SOURCES： An online search of Pubmed and EMCC with computer was applied with the keywords of ＂gene therapy, heart failure＂ to identify related articles published in English from January 1980 to October 2006 and from October 1994 to October 2006. Meanwhile, relevant Chinese articles published between January 1994s and October 2006 were identified in CMCC with the keywords of ＂gene therapy, heart failure＂. STUDY SELECTION： Data were selected primarily, and articles with the trials included above-mentioned treatment group and control group were selected, whereas obvious non-randomized trails were excluded, and the rest literatures were looked for the full text to further judge whether they were RCT according to the inclusion criteria of： （1） The trial contained parallel control groups, i.e. Ordinary pharmacological therapies. （2） Transgenic therapy was adopted in the treatment groups. DATA EXTRACTION： Totally 49 relevant articles of randomized and non-randomized trials were collected, and 31 trials were in accordance with the inclusion criteria, and the others were excluded. DATA SYNTI-IESIS： Thirty-one trials included 478 animal models, which respectively evaluated the patients, underwent different-target tansgene or gene knockout therapies. Of 31 articles 10 did not achieve the anticipated treatment goal, due to the deviation in selection of the aim gene and the transfection carder. CONCLUSION： Although some achievements have been gained in animal experiment, reports of human body gene therapy are not found. With the gene technology progressing, gene therapy is hopefully to become a new treatment of heart failure.