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学科主题: 临床医学
题名:
Updates on Current Advances in Gene Therapy
作者: Tani, Jowy1; Faustine2; Sufian, Jomiany Tani3
关键词: Adenovirus ; gene therapy ; nonviral vectors ; oncolytic viruses ; retrovirus ; suicide gene therapy
刊名: WEST INDIAN MEDICAL JOURNAL
发表日期: 2011-03-01
卷: 60, 期:2, 页:188-194
收录类别: SCI
文章类型: Article
WOS标题词: Science & Technology
类目[WOS]: Medicine, General & Internal
研究领域[WOS]: General & Internal Medicine
关键词[WOS]: PHASE-I TRIAL ; SEVERE COMBINED IMMUNODEFICIENCY ; LEBERS CONGENITAL AMAUROSIS ; DOUBLE-BLIND ; ADENOVIRAL VECTOR ; LENTIVIRAL VECTOR ; GROWTH-FACTOR ; VIRUS ; DISEASE ; DELIVERY
英文摘要:

Gene therapy is the attempt to treat diseases by means of genetic manipulation. Numerous challenges remain to be overcome before it becomes available as a safe and effective treatment option. Retroviruses and adenoviruses are among the most commonly used viral vectors in trials. The retrovirus introduces the gene it carries into the target cell genome while the adenovirus introduces the gene into the target cell nucleus without incorporating it into the target cell genome. Other viral vectors such as adeno-associated viruses, pseudotyped viruses and herpes simplex viruses, are also gaining popularity. Proposed non-viral methods for gene transfer include physical methods and the employment of chemical vectors (lipoplexes, polyplexes and inorganic nanoparticles). Recent studies have investigated potential applications of gene therapy in correcting genetic diseases, treating malignant disorders and for treatment of other diseases. Trials on gene therapy for SCID and Leber′s congenital amaurosis have achieved considerable success, but the widely publicized adverse reaction in X-linked SCID patient receiving gene therapy raised concerns for safety profile of gene therapy.

For that, several methods of improving safety and efficacy of gene therapy have been proposed. At present, the three main gene therapy strategies for treatment of cancer are application to oncolytic viruses, suicide-gene therapy and gene-based immunotherapy. Gendicine, the first approved anticancer drugs based on the use of gene therapy principle, is based on the use of oncolytic viruses. More evidence for wider clinical applications of gene therapy are expected as more gene therapy studies progress from the preclinical phase to clinical trial.

语种: 英语
WOS记录号: WOS:000295239700016
Citation statistics:
内容类型: 期刊论文
URI标识: http://ir.bjmu.edu.cn/handle/400002259/52611
Appears in Collections:北京大学第三临床医学院_期刊论文

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作者单位: 1.Taipei Med Univ, Wanfang Hosp, Dept Neurol, Taipei 116, Taiwan
2.Univ Indonesia, Fac Med, Dr Cipto Mangunkusumo Hosp, Jakarta, Indonesia
3.Peking Univ, Hlth Sci Ctr, Hosp 3, Beijing 100871, Peoples R China

Recommended Citation:
Tani, Jowy,Faustine,Sufian, Jomiany Tani. Updates on Current Advances in Gene Therapy[J]. WEST INDIAN MEDICAL JOURNAL,2011,60(2):188-194.
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