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Neurodegeneration pp 443–455Cite as

Adeno-Associated Viral Gene Delivery in Neurodegenerative Disease

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Part of the book series: Methods in Molecular Biology ((MIMB,volume 793))

Abstract

The advent of viral gene therapy technology has contributed greatly to the study of a variety of medical conditions, and there is increasing promise for clinical translation of gene therapy into human treatments. Adeno-associated viral (AAV) vectors provide one of the more promising approaches to gene delivery, and have been used extensively over the last 20 years. Derived from nonpathogenic parvoviruses, these vectors allow for stable and robust expression of desired transgenes in vitro and in vivo. AAV vectors efficiently and stably transduce neurons, with some strains targeting neurons exclusively in the brain. Thus, AAV vectors are particularly useful for neurodegenerative diseases, which have led to numerous preclinical studies and several human trials of gene therapy in patients with Parkinson’s disease, Alzheimer’s disease, and pediatric neurogenetic disorders. Here, we describe an efficient and reliable method for the production and purification of AAV serotype 2 vectors for both in vitro and in vivo applications.

An erratum to this chapter is available at http://dx.doi.org/10.1007/978-1-61779-328-8_34

An erratum to this chapter can be found at http://dx.doi.org/10.1007/978-1-61779-328-8_34

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Authors and Affiliations

  1. Laboratory of Molecular Neurosurgery, Department of Neurological Surgery, Weill Cornell Medical College, New York, NY, USA

    Peter F. Morgenstern, Roberta Marongiu & Michael G. Kaplitt

  2. Neurologix, Inc., Ft. Lee, NJ, USA

    Sergei A. Musatov

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  1. Peter F. Morgenstern
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  2. Roberta Marongiu
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  3. Sergei A. Musatov
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  4. Michael G. Kaplitt
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Correspondence to Michael G. Kaplitt .

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Editors and Affiliations

  1. Weill Medical College, Dept. Neurology & Neuroscience, Cornell University, East 68th St. 525, New York, 10065, New York, USA

    Giovanni Manfredi

  2. York Avenue 1300, New York, 10065, New York, USA

    Hibiki Kawamata

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Morgenstern, P.F., Marongiu, R., Musatov, S.A., Kaplitt, M.G. (2011). Adeno-Associated Viral Gene Delivery in Neurodegenerative Disease. In: Manfredi, G., Kawamata, H. (eds) Neurodegeneration. Methods in Molecular Biology, vol 793. Humana Press, Totowa, NJ. https://doi.org/10.1007/978-1-61779-328-8_29

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  • DOI: https://doi.org/10.1007/978-1-61779-328-8_29

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