Optimization of AAV expression cassettes to improve packaging capacity and transgene expression in neurons

Author:

Choi Jun-Hyeok,Yu Nam-Kyung,Baek Gi-Chul,Bakes Joseph,Seo Daekwan,Nam Hye Jin,Baek Sung Hee,Lim Chae-Seok,Lee Yong-Seok,Kaang Bong-Kiun

Publisher

Springer Science and Business Media LLC

Subject

Cellular and Molecular Neuroscience,Molecular Biology

Reference27 articles.

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2. Weinberg MS, Samulski RJ, McCown TJ: Adeno-associated virus (AAV) gene therapy for neurological disease. Neuropharmacology. 2013, 69: 82-88.

3. Dong JY, Fan PD, Frizzell RA: Quantitative analysis of the packaging capacity of recombinant adeno-associated virus. Hum Gene Ther. 1996, 7: 2101-2112. 10.1089/hum.1996.7.17-2101.

4. Hermonat PL, Quirk JG, Bishop BM, Han L: The packaging capacity of adeno-associated virus (AAV) and the potential for wild-type-plus AAV gene therapy vectors. FEBS Lett. 1997, 407: 78-84. 10.1016/S0014-5793(97)00311-6.

5. Allocca M, Doria M, Petrillo M, Colella P, Garcia-Hoyos M, Gibbs D, Kim SR, Maguire A, Rex TS, Di Vicino U, Cutillo L, Sparrow JR, Williams DS, Bennett J, Auricchio A: Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in mice. J Clin Invest. 1955–1964, 2008: 118-

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