Gene Therapy: Some History, Applications, Problems, and Prospects
Gene Therapy: Some History, Applications, Problems, and Prospects
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DOI：
10.1177/0192623307309925
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发表时间：
2008-01-01
影响因子：
1.5
通讯作者：
Baum, Bruce J.
中科院分区：
文献类型：
--
作者：
Cotrim, Ana P.;Baum, Bruce J.
将基因转移到组织用于临床的概念已经讨论了近半个世纪，但我们通过重组DNA技术操纵遗传物质的能力使这一目标成为现实。虽然最初被认为是一种治疗常规治疗难以治愈的危及生命的疾病(先天缺陷、癌症)的方法，但现在基因疗法被认为适用于许多非危及生命的疾病，包括那些对患者生活质量产生不利影响的疾病。缺乏合适的治疗成为扩大基因治疗范围的合理基础。本文综述了基因转移的一般方法以及不同的临床应用实例(获得性组织损伤、上胃肠道感染、自身免疫性疾病、全身性蛋白缺乏)。尽管有一些广为人知的问题，基因治疗已经取得了实质性的进展，包括切实的成功，尽管比最初预测的要慢得多。尽管基因治疗仍处于相当原始的阶段，但它有坚实的科学基础。有理由乐观地认为，随着病理生物学理解的增加和生物技术的改进，基因治疗将在20年内成为临床实践的标准部分。
The concept of transferring genes to tissues for clinical applications has been discussed for nearly half a century, but our ability to manipulate genetic material via recombinant DNA technology has brought this goal to reality. While originally conceived as a way to treat life-threatening disorders (inborn errors, cancers) refractory to conventional treatment, gene therapy now is considered for many non-life-threatening conditions, including those adversely affecting a patient's quality of life. The lack of suitable treatment has become a rational basis for extending the scope of gene therapy. This manuscript reviews the general methods by which genes are transferred as well as diverse examples of clinical applications (acquired tissue damage, upper gastrointestinal tract infection, autoimmune disease, systemic protein deficiency). Despite some well-publicized problems, gene therapy has made substantive progress, including tangible success, albeit much slower than was initially predicted. Although gene therapy is still at a fairly primitive stage, it is firmly science based. There is justifiable optimism that with increased pathobiological understanding and biotechnological improvements, gene therapy will become a standard part of clinical practice within 20 years.