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首例人体基因治疗的临床实验迄今虽仅5年,却进展很快。其安全性已为人们所接受。93年全美国已有40例经过批准的临床实验。94年可望达到100余例。其方法简便。给药途径有肌注、静注、皮下注射和局部用药。且治疗一次可长时间地保持在正确的位点表达正常基因,不必经常用药,因而可降低医疗费用。该技术早期使用在遗传性疾病,如囊性纤维变性。用正常透膜电子调节剂通过腺病毒为媒介导入鼻粘膜,获得正常的基因表达,现已治疗多种遗传病,如视网膜色素变性聋哑综合征、甲状腺肿耳聋综合征等。
The first case of human gene therapy clinical trials so far, only 5 years, but progress is fast. Its safety has been accepted by people. In 1993, there were 40 approved clinical trials in the United States. 94 years is expected to reach more than 100 cases. The method is simple. Routes of administration include intramuscular injection, intravenous injection, subcutaneous injection and topical application. And treatment time can be maintained at the right place for a long time to express normal genes, without frequent medication, which can reduce medical costs. The technology was used early in hereditary diseases such as cystic fibrosis. With normal transmembrane electronic regulator through the adenovirus as a medium into the nasal mucosa to obtain normal gene expression, has been treated a variety of genetic diseases, such as dementia retina pigmented degeneration syndrome, goiter deafness syndrome.