Agarwal S, Khardori R, Khardori N
Department of Genetics, Sanjay Gandhi Post Graduate Institute of Medical Sciences, Lucknow.
J Assoc Physicians India. 1992 Feb;40(2):95-8.
Gene therapy is a way to treat disease by transfer of genetic material into the cells of a diseased organism. Generally it is transferred in the form of one or few genes. There is a tremendous potential for application of this technique to the treatment of human hereditary diseases, particularly single gene disorders. Due to ethical and practical reasons, it is currently being used only as somatic cell therapy but it could be achieved by germline also. The major problem in gene therapy is achieving an efficient gene transfer and a persistent gene expression in appropriate somatic cells.
基因治疗是一种通过将遗传物质转移到患病生物体的细胞中来治疗疾病的方法。一般来说，它是以一个或几个基因的形式进行转移的。这项技术在治疗人类遗传性疾病，特别是单基因疾病方面具有巨大的应用潜力。由于伦理和实际原因，目前它仅作为体细胞治疗使用，但也可以通过生殖系实现。基因治疗中的主要问题是在适当的体细胞中实现高效的基因转移和持续的基因表达。
