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Targeted gene repair: from RNA/DNA to single-stranded oligonucleotides

机译：靶向基因修复：从RNA / DNA到单链寡核苷酸

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Sequencing of the human genome, understanding the genetic basis of inherited and acquired disorders and the dramatic technological innovations in molecular biology, have provided the fundamental tools to cure many diseases by genetic manipulation Two gene therapy approaches relevant to the treatment of inherited dysfunctions are gene augmentation and gene repair. Gene augmentation compensates for a defective gene by introducing a functional transgene, and thus can treat recessive disorders Some viruses, such as adeno-, adeno-associated, lenti- and retroviruses have been modified to act as delivery vectors for a transgene. Each viral vector has its own distinctive features for specificity of transduction and integration, and this results in both their advantages and limitations. For example, adenoviruses can efficiently infect non-dividing cells but cannot integrate the transgene into the host genome. In contrast, retroviral vectors can integrate their cargo only in proliferating cells, Despite being the most common gene therapy approach, viral vectors have considerable safety and specificity issues that must be resolved prior to routine clinical use. Their pathogenicity, immunogenicity, potential insertional mutagenesis and/or onco-genesis, induction of epigenetic changes and transgene silencing are just some of the potential challenges for use in gene therapy (for reviews see refs 1 and 2),,

机译：人类基因组的测序，了解遗传和获得的疾病的遗传基础以及分子生物学中的戏剧性技术创新，提供了通过遗传操作治疗许多疾病的基本工具，这两个基因治疗方法与遗传功能障碍的治疗方法是基因增强和基因修复。基因增强通过引入功能转基因来补偿缺陷的基因，因此可以治疗隐性疾病一些病毒，例如腺样，腺相关，氟和逆转录病毒，以作为转基因的递送载体。每个病毒载体都有自己的独特特征，用于转导和集成的特异性，这导致它们的优缺点和局限性。例如，腺病毒可以有效地感染非分配细胞，但不能将转基因与宿主基因组相容。相比之下，逆转录病毒载体可以仅在增殖细胞中整合其货物，尽管是最常见的基因治疗方法，病毒载体具有相当大的安全性和特异性问题，必须在常规临床使用前解决。它们的致病性，免疫原性，潜在的插入诱变和/或onGo-Genesis，表观遗传变化和转基因沉默的诱导只是在基因治疗中使用的一些潜在挑战（用于参考文献1和2）,,

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《Falk Symposium》|2004年||共24页
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W. YIN; B. T. KREN; C. J. STEER;
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1. In vivo gene repair of point and frameshift mutations directed by chimeric RNA/DNA oligonucleotides and modified single-stranded oligonucleotides [J] . Liu L, Rice MC, Kmiec EB Nucleic Acids Research . 2001,第20期

机译：嵌合RNA / DNA寡核苷酸和修饰的单链寡核苷酸指导的点和移码突变的体内基因修复
2. In vivo gene repair of point and frameshift mutations directed by chimeric RNA/DNA oligonucleotides and modified single-stranded oligonucleotides [J] . Eric B. Kmiec, Li Liu, Michael C. Rice Nucleic acids research . 2001,第20期

机译：嵌合RNA / DNA寡核苷酸和修饰的单链寡核苷酸指导的点和移码突变的体内基因修复
3. Specific targeted gene repair using single-stranded DNA oligonucleotides at an endogenous locus in mammalian cells uses homologous recombination. [J] . McLachlan J, Fernandez S, Helleday DNA repair . 2009,第12期

机译：在哺乳动物细胞的内源性位点使用单链DNA寡核苷酸进行特异性靶向基因修复，使用同源重组。
4. Targeted gene repair: from RNA/DNA to single-stranded oligonucleotides [C] . W. YIN, B. T. KREN, C. J. STEER Falk Symposium . 2004

机译：靶向基因修复：从RNA / DNA到单链寡核苷酸
5. Therapeutic single-stranded oligonucleotides in gene repair and cancer. [D] . Schwartz, Timothy R. 2008

机译：基因修复和癌症中的治疗性单链寡核苷酸。
6. In vivo gene repair of point and frameshift mutations directed by chimeric RNA/DNA oligonucleotides and modified single-stranded oligonucleotides [O] . Li Liu, Michael C. Rice, Eric B. Kmiec 2001

机译：嵌合RNA / DNA寡核苷酸和修饰的单链寡核苷酸指导的点和移码突变的体内基因修复
7. In vivo gene repair of point and frameshift mutations directed by chimeric RNA/DNA oligonucleotides and modified single-stranded oligonucleotides [O] . Liu, Li, Rice, Michael C., Kmiec, Eric B. 2001

机译：嵌合RNA / DNA寡核苷酸和修饰的单链寡核苷酸指导的点和移码突变的体内基因修复

Targeted gene repair: from RNA/DNA to single-stranded oligonucleotides

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