Hori Tesshu, Fukutome Masashi, Koike Chieko
Laboratory for Systems Neuroscience and Developmental Biology, College of Pharmaceutical Sciences, Ritsumeikan University.
Graduate School of Life Sciences, Ritsumeikan University.
Biol Pharm Bull. 2019;42(3):343-347. doi: 10.1248/bpb.b18-00913.
With an increasing number of identified causative genes, the widespread use of gene therapy is quickly becoming feasible. Once a target gene is selected, it is important to have a cell delivery method that is both specific and efficient. Cell type specificity and high efficiency is particularly important for the treatment of retinal degeneration, since viruses are efficient gene delivery vehicles for the nervous system, but often bring with them non-specific infections. In this review, we focus on adeno-associated virus (AAV). Over the last few decades, AAV has become a leading choice for safe gene delivery, in part due to its replication deficiency in cells without a helper virus. Here, we summarize the tropism of recombinant AAV (rAAV) for various types of mammalian retinal neurons in relation to capsid serotype and administration method. We also include our recent findings on an AAV serotype that AAV was specifically infected mouse cone photoreceptors when delivered by subretinal administration.
随着越来越多的致病基因被确定，基因治疗的广泛应用正迅速变得可行。一旦选择了目标基因，拥有一种既特异又高效的细胞递送方法就很重要。细胞类型特异性和高效性对于视网膜变性的治疗尤为重要，因为病毒是神经系统高效的基因递送载体，但常常会带来非特异性感染。在这篇综述中，我们聚焦于腺相关病毒（AAV）。在过去几十年里，AAV已成为安全基因递送的首选，部分原因是它在没有辅助病毒的细胞中缺乏复制能力。在这里，我们总结了重组腺相关病毒（rAAV）与衣壳血清型和给药方法相关的对各种类型哺乳动物视网膜神经元的嗜性。我们还纳入了我们最近的发现，即一种AAV血清型通过视网膜下给药时能特异性感染小鼠视锥光感受器。
