Skubis-Zegadło Joanna, Stachurska Anna, Małecki Maciej
Department of Applied Pharmacy and Bioengineering, Medical University of Warsaw, Banacha 1, 02-097 Warsaw, Poland, 0048 572-09-65,
Med Wieku Rozwoj. 2013 Jul-Sep;17(3):202-6.
In vivo gene replacement is one of the most compelling concepts in modern medicine. Adeno-associated virus (AAV) vectors are currently among the most frequently used viral vectors for gene therapy and they have shown therapeutic efficacy in a range of animal models. The lack of pathogenicity of the virus, low immunogenicity, its stability, and many available serotypes have increased AAV's potential as a delivery vehicle for gene therapy applications. There are some limitations to the use of rAAV in gene therapy. The first is their size. Due to the small size of the vector, the ability to conduct a therapeutic gene expression cassette is limited. Another limitation is the common occurrence of neutralizing antibodies in human populations. This review will focus on the biology of AAV, its use as a vector for gene therapy and mechanisms of AAV/host cell interaction.
体内基因替代是现代医学中最具吸引力的概念之一。腺相关病毒（AAV）载体是目前基因治疗中最常用的病毒载体之一，并且它们已在一系列动物模型中显示出治疗效果。该病毒缺乏致病性、免疫原性低、稳定性好以及有多种可用血清型，这些都增加了AAV作为基因治疗应用递送载体的潜力。在基因治疗中使用重组AAV存在一些局限性。首先是它们的大小。由于载体尺寸小，进行治疗性基因表达盒的能力有限。另一个局限性是人群中中和抗体的普遍存在。本综述将重点关注AAV的生物学特性、其作为基因治疗载体的用途以及AAV/宿主细胞相互作用的机制。
