Recombinant adeno-associated virus: Clinical application and development as a gene-therapy vector

XiaoP.-J.; LentzT.B.; SamulskiR.J.

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Recombinant adeno-associated virus: Clinical application and development as a gene-therapy vector

机译：重组腺伴随病毒：作为基因治疗载体的临床应用和发展

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Gene therapy is gaining momentum as a method of treating human disease. Initially conceived as a strategy to complement defective genes in monogenic disorders, the scope of gene therapy has expanded to encompass a variety of applications. Likewise, the molecular tools for gene delivery have evolved and diversified to meet these various therapeutic needs. Recombinant adeno-associated virus (rAAV) has made significant strides toward clinical application with an excellent safety profile and successes in several clinical trials. This review covers the basic biology of rAAV as a gene therapy vector as well as its advantages compared with other methods of gene delivery. The status of clinical trials utilizing rAAV is also discussed in detail. In conclusion, methods of engineering the vector to overcome challenges identified from these trials are covered, with emphasis on modification of the viral capsid to increase the tissue/cell-specific targeting and transduction efficiency.

机译：基因疗法作为治疗人类疾病的一种方法正获得发展。最初被认为是对单基因疾病中的缺陷基因进行补充的一种策略，但基因治疗的范围已扩展到涵盖各种应用。同样，用于基因递送的分子工具已经发展并多样化，以满足这些各种治疗需求。重组腺相关病毒（rAAV）已以卓越的安全性和在多项临床试验中的成功向临床应用迈进了一大步。这篇综述涵盖了rAAV作为基因治疗载体的基本生物学原理，以及与其他基因传递方法相比的优势。还详细讨论了利用rAAV进行临床试验的情况。总之，涵盖了工程化载体以克服这些试验中确定的挑战的方法，重点是修饰病毒衣壳以增加组织/细胞特异性靶向和转导效率。

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《Therapeutic delivery》 |2012年第7期|共22页
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XiaoP.-J.; LentzT.B.; SamulskiR.J.;
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正文语种 eng
中图分类临床医学;
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1. Recombinant adeno-associated virus: Clinical application and development as a gene-therapy vector [J] . XiaoP.-J., LentzT.B., SamulskiR.J. Therapeutic delivery . 2012,第7期

机译：重组腺伴随病毒：作为基因治疗载体的临床应用和发展
2. Strategies for manufacturing recombinant adeno-associated virus vectors for gene therapy applications exploiting baculovirus technology [J] . Alejandro Negret, Robert M. Kotin Briefings in functional genomics & proteomics . 2008,第4期

机译：利用杆状病毒技术生产用于基因治疗的重组腺相关病毒载体的策略
3. Strategies for manufacturing recombinant adeno-associated virus vectors for gene therapy applications exploiting baculovirus technology [J] . Alejandro NegreteAlejandro Negrete holds a PhD in Chemical Engineering from Birmingham University UK. As a postdoctoral researcher at the NIH he focuses in process development for manufacture gene therapy viral vectors. and Robert M. KotinRobert Kotin has been active in adeno-associated virus (AAV) research since receiving his PhD in 1986. As a post-doctoral fellow he discovered that the genome of AAV site specifically integrates into human chromosome 19. He has been at the NIH since 1994. His paper describing AAV production using baculovirus—insect cell technology was published in 2002. Briefings in Functional Genomics & Proteomics . 2008,第4期

机译：利用杆状病毒技术生产用于基因治疗的重组腺相关病毒载体的策略
4. Effects of phospholamban antisense RNA on SERCA and CaI in rat cardiomyocytes using recombinant adeno-associated virus vector [C] . LI Jiang, HU Shen-jiang, SUN Jian, The 2nd World Congress for Chinese Biomedical Engineers . 2004

机译：重组腺相关病毒载体对磷酸lamban反义RNA对大鼠心肌细胞SERCA和Ca I的影响
5. Ongoing cellular responses to transgene products encoded by recombinant adeno-associated virus (RAAV) vectors. [D] . Best, Victoria. 2010

机译：对重组腺相关病毒（RAAV）载体编码的转基因产物的持续细胞应答。
6. Strategies for manufacturing recombinant adeno-associated virus vectors for gene therapy applications exploiting baculovirus technology [O] . Alejandro Negrete, Robert M. Kotin -1

机译：利用杆状病毒技术生产用于基因治疗的重组腺相关病毒载体的策略
7. Production of Recombinant Adeno-associated Virus Vectors Using Suspension HEK293 Cells and Continuous Harvest of Vector From the Culture Media for GMP FIX and FLT1 Clinical Vector [O] . Joshua C Grieger, Stephen M Soltys, Richard Jude Samulski 2016

机译：使用悬浮HEK293细胞的重组腺相关病毒载体的生产，并从培养介质中连续收获来自GMP FIX和FLT1临床载体
8. Development of Targeted Sindbis Virus Vectors for Potential Application to Breast Cancer Therapy [R] . Dropulic, L. K. 2003

机译：靶向辛德毕斯病毒载体的开发可用于乳腺癌治疗

Recombinant adeno-associated virus: Clinical application and development as a gene-therapy vector

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