Adeno-associated viral vectors have numerous applications in neuroscience, including the study of gene function in health and disease, targeting of light-sensitive proteins to anatomically distinct sets of neurons to manipulate neuronal activity (optogenetics), and the delivery of fluorescent protein to study anatomical connectivity in the brain. Moreover several phase I/II clinical trials for gene therapy of eye and brain diseases with adeno-associated viral vectors have shown that these vectors are well tolerated by human patients. In this chapter we describe a detailed protocol for the small scale production of recombinant adeno-associated viral vectors. This protocol can be executed by investigators with experience in cell culture and molecular biological techniques in any well-equipped molecular neurobiology laboratory. With this protocol we typically obtain research batches of 100–200 μL that range in titer from 5 × 1012 to 2 × 1013 genomic copies/mL.
CITATION STYLE
Verhaagen, J., Hobo, B., Ehlert, E. M. E., Eggers, R., Korecka, J. A., Hoyng, S. A., … Mason, M. R. J. (2018). Small scale production of recombinant adeno-associated viral vectors for gene delivery to the nervous system. In Methods in Molecular Biology (Vol. 1715, pp. 3–17). Humana Press Inc. https://doi.org/10.1007/978-1-4939-7522-8_1
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