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单词 Somatic gene therapy
释义

Somatic gene therapy

中文百科

基因治疗 Gene therapy

(重定向自Somatic gene therapy)
基因治疗用腺病毒载体。使用腺病毒的新基因插入细胞。如果治疗是成功的,新的基因,将产生功能性蛋白来治疗疾病。

基因治疗基因疗法英语:gene therapy)是利用分子生物学方法将目的基因导入患者体内,使之表达目的基因产物,从而使疾病得到治疗,为现代医学和分子生物学相结合而诞生的新技术。基因治疗作为疾病治疗的新手段,它已有一些成功的应用,并且科学突破将继续推动基因治疗向主流医疗发展。

科学家们由逻辑推理出合理步骤,尝试将基因直接植入人体细胞中,其中,重点关注一些由单基因缺陷引起的疾病,如囊肿性纤维化,血友病,肌肉萎缩症和镰状细胞性贫血。不过,由于携带大段DNA并将其置入基因组的正确的位置非常困难,这种技术没有得到普及。今天,大多数的基因治疗研究的目的都是弥补癌症和遗传疾病的基因缺陷或丢失。

英语百科

Gene therapy 基因治疗

(重定向自Somatic gene therapy)
Gene therapy using an adenovirus vector. In some cases, the adenovirus will insert the new gene into a cell. If the treatment is successful, the new gene will make a functional protein to treat a disease.
A duplex of crRNA and tracrRNA acts as guide RNA to introduce a specifically located gene modification based on the RNA 5’ upstream of the crRNA. Cas9 binds the tracrRNA and needs a DNA binding sequence (5’NGG3’), which is called protospacer adjacent motif (PAM). After binding, Cas9 introduces a DNA double strand break, which is then followed by gene modification via homologous recombination (HDR) or non-homologous end joining (NHEJ).

Gene therapy is the therapeutic delivery of nucleic acid polymers into a patient's cells as a drug to treat disease.

The origins of gene therapy can be traced back to the first live attenuated vaccines in the 1950s. Although attenuated vaccines do not alter extant human genes, viruses are RNA polymers with their own genetic code that acts upon human cells, thus live vaccines can be considered a primitive form of gene therapy, albeit not in the sense that is generally implied today.

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更新时间:2025/6/17 9:25:30