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基因治疗的基础科学。

The basic science of gene therapy.

作者信息

Mulligan R C

机构信息

Whitehead Institute for Biomedical Research, Massachusetts Institute of Technology, Cambridge 02142.

出版信息

Science. 1993 May 14;260(5110):926-32. doi: 10.1126/science.8493530.

DOI:10.1126/science.8493530
PMID:8493530
Abstract

The development over the past decade of methods for delivering genes to mammalian cells has stimulated great interest in the possibility of treating human disease by gene-based therapies. However, despite substantial progress, a number of key technical issues need to be resolved before gene therapy can be safely and effectively applied in the clinic. Future technological developments, particularly in the areas of gene delivery and cell transplantation, will be critical for the successful practice of gene therapy.

摘要

在过去十年中,将基因导入哺乳动物细胞的方法取得了进展,这激发了人们对通过基因疗法治疗人类疾病可能性的极大兴趣。然而,尽管取得了重大进展,但在基因疗法能够安全有效地应用于临床之前,仍有一些关键技术问题需要解决。未来的技术发展,尤其是在基因递送和细胞移植领域,对于基因疗法的成功应用至关重要。

相似文献

1
The basic science of gene therapy.基因治疗的基础科学。
Science. 1993 May 14;260(5110):926-32. doi: 10.1126/science.8493530.
2
[Gene therapy].[基因治疗]
Rev Invest Clin. 1997 Sep-Oct;49(5):397-409.
3
[Gene therapy: new developments].[基因治疗:新进展]
Praxis (Bern 1994). 2002 Dec 18;91(51-52):2227-35. doi: 10.1024/0369-8394.91.51.2227.
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Progress towards the clinical application of helper-dependent adenoviral vectors for liver and lung gene therapy.用于肝脏和肺部基因治疗的辅助依赖型腺病毒载体临床应用的进展。
Curr Opin Mol Ther. 2006 Oct;8(5):446-54.
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Retroviral gene therapy in hematopoietic diseases.造血系统疾病的逆转录病毒基因治疗。
J Clin Apher. 1997;12(4):187-93. doi: 10.1002/(sici)1098-1101(1997)12:4<187::aid-jca6>3.0.co;2-6.
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[Gene therapy for liver diseases].[肝脏疾病的基因治疗]
Gastroenterol Hepatol. 2000 Oct;23(8):394-402.
7
Progress towards liver and lung-directed gene therapy with helper-dependent adenoviral vectors.用辅助依赖性腺病毒载体实现肝脏和肺部靶向基因治疗的进展。
Curr Gene Ther. 2009 Oct;9(5):329-40. doi: 10.2174/156652309789753310.
8
Gene transfer and the prospects for somatic gene therapy.基因转移与体细胞基因治疗的前景。
Hematol Oncol Clin North Am. 1988 Jun;2(2):277-87.
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[Constitutional hematologic diseases. Perspectives of gene therapy].[体质性血液疾病。基因治疗的前景]
Presse Med. 1991 Jan 26;20(3):117-23.
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Gene transfer in the treatment of hematologic disease.基因转移在血液疾病治疗中的应用。
Exp Hematol. 1990 Aug;18(7):857-60.

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