靶向基因修复——进入实战阶段。
Targeted gene repair -- in the arena.
作者信息
Kmiec Eric B
机构信息
Department of Biology, Delaware Biotechnology Institute, University of Delaware, Newark, Delaware 19716, USA.
出版信息
J Clin Invest. 2003 Sep;112(5):632-6. doi: 10.1172/JCI19777.
Abstract
The development of targeted gene repair is under way and, despite some setbacks, shows promise as an alternative form of gene therapy. This approach uses synthetic DNA molecules to activate and direct the cell's inherent DNA repair systems to correct inborn errors. The progress of this technique and its therapeutic potential are discussed in relation to the treatment of genetic diseases.
摘要
靶向基因修复技术正在发展，尽管遇到了一些挫折，但作为一种基因治疗的替代形式显示出了前景。这种方法使用合成DNA分子来激活并引导细胞固有的DNA修复系统，以纠正先天性错误。本文结合遗传病的治疗探讨了该技术的进展及其治疗潜力。
相似文献
1
Targeted gene repair -- in the arena.靶向基因修复——进入实战阶段。
J Clin Invest. 2003 Sep;112(5):632-6. doi: 10.1172/JCI19777.
2
Gene therapy progress and prospects: targeted gene repair.基因治疗的进展与前景：靶向基因修复
Gene Ther. 2005 Apr;12(8):639-46. doi: 10.1038/sj.gt.3302511.
3
The development and regulation of gene repair.基因修复的发展与调控
Nat Rev Genet. 2003 Sep;4(9):679-89. doi: 10.1038/nrg1156.
4
Targeted gene repair in mammalian cells using chimeric RNA/DNA oligonucleotides and modified single-stranded vectors.使用嵌合RNA/DNA寡核苷酸和修饰的单链载体在哺乳动物细胞中进行靶向基因修复。
Sci STKE. 2001 Mar 13;2001(73):pl1. doi: 10.1126/stke.2001.73.pl1.
5
Recent developments and perspectives on gene therapy using synthetic vectors.使用合成载体进行基因治疗的最新进展与展望。
Ther Deliv. 2013 Jan;4(1):95-113. doi: 10.4155/tde.12.128.
6
Split vector systems for ultra-targeted gene delivery: a contrivance to achieve ethical assurance of somatic gene therapy in vivo.用于超靶向基因递送的分裂载体系统：一种在体内实现体细胞基因治疗伦理保障的策略。
Med Hypotheses. 2014 Aug;83(2):211-6. doi: 10.1016/j.mehy.2014.04.027. Epub 2014 May 2.
7
Gene therapy.基因治疗。
Handb Clin Neurol. 2012;104:331-8. doi: 10.1016/B978-0-444-52138-5.00021-9.
8
Use of gene targeting to study recombination in mammalian cell DNA repair mutants.利用基因靶向技术研究哺乳动物细胞DNA修复突变体中的重组。
Methods Mol Biol. 2012;920:445-70. doi: 10.1007/978-1-61779-998-3_31.
9
Non-viral vectors in cystic fibrosis gene therapy: progress and challenges.囊性纤维化基因治疗中的非病毒载体：进展与挑战
Trends Biotechnol. 2004 Nov;22(11):586-92. doi: 10.1016/j.tibtech.2004.09.009.
10
The potential of gene transfer for the treatment of osteoarthritis.基因转移在骨关节炎治疗中的潜力。
Regen Med. 2014 Jan;9(1):5-8. doi: 10.2217/rme.13.70.
引用本文的文献
1
Prevention of Transcriptional γ-globin Gene Silencing by Inducing The Hereditary Persistence of Fetal Hemoglobin Point Mutation Using Chimeraplast-Mediated Gene Targeting.通过嵌合质粒介导的基因靶向诱导胎儿血红蛋白点突变的遗传性持续来预防转录性γ-珠蛋白基因沉默
Cell J. 2018 Oct;20(3):318-325. doi: 10.22074/cellj.2018.5181. Epub 2018 May 15.
2
Genetic spell-checking: gene editing using single-stranded DNA oligonucleotides.基因拼写检查：使用单链DNA寡核苷酸进行基因编辑。
Plant Biotechnol J. 2016 Feb;14(2):463-70. doi: 10.1111/pbi.12473. Epub 2015 Sep 24.
3
Somatic correction of junctional epidermolysis bullosa by a highly recombinogenic AAV variant.通过一种高度重组的腺相关病毒变体对交界性大疱性表皮松解症进行体细胞矫正。
Mol Ther. 2014 Apr;22(4):725-33. doi: 10.1038/mt.2013.290. Epub 2014 Jan 6.
4
RecA-independent single-stranded DNA oligonucleotide-mediated mutagenesis.不依赖RecA的单链DNA寡核苷酸介导的诱变
F1000 Biol Rep. 2010 Jul 22;2:56. doi: 10.3410/B2-56.
5
Characterization of an MPS I-H knock-in mouse that carries a nonsense mutation analogous to the human IDUA-W402X mutation.一种 MPS I-H 基因敲入小鼠的特征，该小鼠携带一种类似于人类 IDUA-W402X 突变的无义突变。
Mol Genet Metab. 2010 Jan;99(1):62-71. doi: 10.1016/j.ymgme.2009.08.002.
6
The involvement of replication in single stranded oligonucleotide-mediated gene repair.复制在单链寡核苷酸介导的基因修复中的作用。
Nucleic Acids Res. 2006;34(21):6183-94. doi: 10.1093/nar/gkl852. Epub 2006 Nov 6.
7
Correction of chromosomal mutation and random integration in embryonic stem cells with helper-dependent adenoviral vectors.利用辅助依赖型腺病毒载体纠正胚胎干细胞中的染色体突变和随机整合。
Proc Natl Acad Sci U S A. 2005 Sep 20;102(38):13628-33. doi: 10.1073/pnas.0506598102. Epub 2005 Sep 7.
8
Increased efficiency of oligonucleotide-mediated gene repair through slowing replication fork progression.通过减缓复制叉进展提高寡核苷酸介导的基因修复效率。
Proc Natl Acad Sci U S A. 2005 Feb 15;102(7):2508-13. doi: 10.1073/pnas.0406991102. Epub 2005 Feb 4.
9
Targeted genetic repair: an emerging approach to genetic therapy.靶向基因修复：一种新兴的基因治疗方法。
J Clin Invest. 2003 Aug;112(3):310-1. doi: 10.1172/JCI19419.
本文引用的文献
1
Nuclease activity of Saccharomyces cerevisiae Mre11 functions in targeted nucleotide alteration.酿酒酵母Mre11的核酸酶活性在靶向核苷酸改变中发挥作用。
Appl Environ Microbiol. 2003 Oct;69(10):6216-24. doi: 10.1128/AEM.69.10.6216-6224.2003.
2
Efficiency of chimeraplast gene targeting by direct nuclear injection using a GFP recovery assay.利用绿色荧光蛋白恢复试验通过直接核注射进行嵌合质粒基因靶向的效率
Mol Ther. 2003 Feb;7(2):248-53. doi: 10.1016/s1525-0016(02)00039-4.
3
Rad51p and Rad54p, but not Rad52p, elevate gene repair in Saccharomyces cerevisiae directed by modified single-stranded oligonucleotide vectors.Rad51p和Rad54p，但不是Rad52p，可提高酿酒酵母中由修饰的单链寡核苷酸载体指导的基因修复。
Nucleic Acids Res. 2002 Jul 1;30(13):2742-50. doi: 10.1093/nar/gkf397.
4
The 'biased' evolution of targeted gene repair.靶向基因修复的“偏向性”进化
Curr Opin Mol Ther. 2002 Apr;4(2):171-6.
5
Strand bias in targeted gene repair is influenced by transcriptional activity.靶向基因修复中的链偏向受转录活性影响。
Mol Cell Biol. 2002 Jun;22(11):3852-63. doi: 10.1128/MCB.22.11.3852-3863.2002.
6
Dystrophin gene repair in mdx muscle precursor cells in vitro and in vivo mediated by RNA-DNA chimeric oligonucleotides.RNA-DNA嵌合寡核苷酸介导的体外和体内mdx肌肉前体细胞中的肌营养不良蛋白基因修复。
Hum Gene Ther. 2002 Apr 10;13(6):707-18. doi: 10.1089/104303402317322276.
7
Targeted gene repair in mammalian cells using chimeric RNA/DNA oligonucleotides and modified single-stranded vectors.使用嵌合RNA/DNA寡核苷酸和修饰的单链载体在哺乳动物细胞中进行靶向基因修复。
Sci STKE. 2001 Mar 13;2001(73):pl1. doi: 10.1126/stke.2001.73.pl1.
8
In vivo gene repair of point and frameshift mutations directed by chimeric RNA/DNA oligonucleotides and modified single-stranded oligonucleotides.由嵌合RNA/DNA寡核苷酸和修饰的单链寡核苷酸引导的点突变和移码突变的体内基因修复。
Nucleic Acids Res. 2001 Oct 15;29(20):4238-50. doi: 10.1093/nar/29.20.4238.
9
Targeted correction of the point mutations of beta-thalassemia and targeted mutagenesis of the nucleotide associated with HPFH by RNA/DNA oligonucleotides: potential for beta-thalassemia gene therapy.通过RNA/DNA寡核苷酸对β地中海贫血的点突变进行靶向校正以及对与遗传性胎儿血红蛋白持续存在症（HPFH）相关的核苷酸进行靶向诱变：β地中海贫血基因治疗的潜力
Blood Cells Mol Dis. 2001 Mar-Apr;27(2):530-8. doi: 10.1006/bcmd.2001.0416.
10
In vitro and in vivo nucleotide exchange directed by chimeric RNA/DNA oligonucleotides in Saccharomyces cerevisae.酿酒酵母中嵌合RNA/DNA寡核苷酸介导的体外和体内核苷酸交换
Mol Microbiol. 2001 May;40(4):857-68. doi: 10.1046/j.1365-2958.2001.02407.x.
Zotero 插件