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据Eckardt S 2011年1月10日(J Clin Invest,2001 Jan 10.pii:45377.doi:10.1172/JCI45377.[Epub ahead of print])报道,利用生殖细胞产生胚胎干细胞,可解决遗传疾病的问题,该方法与无需基因组工程的典型基因治疗方法不同。美国全国儿童医院研究所研究人员公布了一份基因治疗方案,改善鼠实验中的遗传性血液紊乱——β-地中海贫血,基因改善是使用鼠的未受精卵制造胚胎干细胞系。这些干细胞系没有遗传疾病基因,可用于小鼠的移植治疗。对于治疗常染色体显性遗传
According to Eckardt S, Jan. 10, 2011 (J Clin Invest, 2001 Jan. 10.pii: 45377. doi: 10.1172 / JCI45377. [Epub ahead of print]), the use of germ cells to generate embryonic stem cells can solve the problem of genetic diseases , This method is different from the typical gene therapy method that does not require genomic engineering. Researchers at the National Institutes of the Children’s Hospital of the United States have released a gene therapy program to improve hereditary blood disorders in rat experiments - beta-thalassemia - genetic improvement using mouse uninoculated eggs to create embryonic stem cell lines. These stem cell lines have no genetic disease genes that can be used in mouse transplantations. For the treatment of autosomal dominant inheritance