Flannery John G, Visel Meike
Neuroscience Division, Department of Molecular and Cell Biology, Helen Wills Neuroscience Institute, University of California, Berkeley, CA, USA.
Methods Mol Biol. 2013;935:351-69. doi: 10.1007/978-1-62703-080-9_25.
Adeno-associated virus (AAV) vectors are in wide use for in vivo gene transfer for the treatment of inherited retinal disease. AAV vectors have been tested in many animal models and have demonstrated efficacy with low toxicity. In this chapter we describe some of the recent methods for small-scale production of these vectors for use in a laboratory setting in volumes and purity appropriate for testing in small and large animals.
腺相关病毒（AAV）载体被广泛用于体内基因转移，以治疗遗传性视网膜疾病。AAV载体已在许多动物模型中进行了测试，并显示出低毒性的有效性。在本章中，我们描述了一些近期用于小规模生产这些载体的方法，这些方法适用于实验室环境，所生产的载体的量和纯度适合在小型和大型动物中进行测试。
