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基因治疗:早期的前景、后续的问题及近期的突破

Gene therapy, early promises, subsequent problems, and recent breakthroughs.

作者信息

Razi Soofiyani Saeideh, Baradaran Behzad, Lotfipour Farzaneh, Kazemi Tohid, Mohammadnejad Leila

机构信息

Drug Applied Research Center, Tabriz University of Medical Sciences, Tabriz, Iran. ; Immonuology Research Center, Tabriz University of Medical Sciences, Tabriz, Iran.

出版信息

Adv Pharm Bull. 2013;3(2):249-55. doi: 10.5681/apb.2013.041. Epub 2013 Aug 20.

Abstract

Gene therapy is one of the most attractive fields in medicine. The concept of gene delivery to tissues for clinical applications has been discussed around half a century, but scientist's ability to manipulate genetic material via recombinant DNA technology made this purpose to reality. Various approaches, such as viral and non-viral vectors and physical methods, have been developed to make gene delivery safer and more efficient. While gene therapy initially conceived as a way to treat life-threatening disorders (inborn errors, cancers) refractory to conventional treatment, to date gene therapy is considered for many non-life-threatening conditions including those adversely influence on a patient's quality of life. Gene therapy has made significant progress, including tangible success, although much slower than was initially predicted. Although, gene therapies still at a fairly primitive stage, it is firmly science based. There is justifiable hope that with enhanced pathobiological understanding and biotechnological improvements, gene therapy will be a standard part of clinical practice within 20 years.

摘要

基因治疗是医学领域中最具吸引力的领域之一。将基因递送至组织用于临床应用的概念已被讨论了大约半个世纪,但科学家通过重组DNA技术操纵遗传物质的能力使这一目标成为现实。为了使基因递送更安全、更高效,人们开发了各种方法,如病毒载体和非病毒载体以及物理方法。虽然基因治疗最初被设想为一种治疗传统治疗难以治愈的危及生命的疾病(先天性疾病、癌症)的方法,但到目前为止,基因治疗已被应用于许多非危及生命的疾病,包括那些对患者生活质量有不利影响的疾病。基因治疗已经取得了重大进展,包括切实的成功,尽管进展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。有理由希望,随着对病理生物学的深入理解和生物技术的改进,基因治疗将在20年内成为临床实践的标准组成部分。

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本文引用的文献

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