Rossi Axel, Salvetti Anna
Centre international de recherche en infectiologie (CIRI), Inserm U1111, CNRS UMR5308, équipe NucléoVir, École normale supérieure de Lyon, 46, allée d'Italie, 69007 Lyon, France.
Med Sci (Paris). 2016 Feb;32(2):167-74. doi: 10.1051/medsci/20163202010. Epub 2016 Mar 2.
Recombinant AAV vectors (rAAV) are considered as very efficient tools for in vivo gene transfer. Accordingly, several preclinical and clinical gene therapy trials use these vectors to treat inherited and acquired diseases. rAAV vectors possess the capacity to persist for a long term in the transduced tissue in a transcriptionally active, extra-chromosomal (episomal) form. However, many studies have shown that a significant fraction of the rAAV genomes can also nonspecifically integrate into the host cell genome thus raising the possibility of insertional mutagenesis events. This review summarizes the current knowledge on integration of wild type and rAAV genomes and highlights the major questions which remain unresolved.
重组腺相关病毒载体（rAAV）被认为是体内基因转移的非常有效的工具。因此，一些临床前和临床基因治疗试验使用这些载体来治疗遗传性和获得性疾病。rAAV载体能够以转录活性的、染色体外（游离）形式在转导组织中长期持续存在。然而，许多研究表明，相当一部分rAAV基因组也能非特异性地整合到宿主细胞基因组中，从而增加了插入诱变事件的可能性。本综述总结了关于野生型和rAAV基因组整合的当前知识，并强调了仍未解决的主要问题。
