By T.R. Flotte, and K.I. Berns (Eds.)
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Extra info for Adeno-Associated Viral Vectors for Gene Therapy
1983). Nucleotide sequence and organization of the adeno‐associated virus 2 genome. J. Virol. 45, 555–564. , Wilson, J. , Kleckner, A. L. and Mendell, J. (2000). Phase I clinical trial utilizing gene therapy for limb girdle muscular dystrophy: Alpha‐, beta‐, gamma‐, or delta‐sarcoglycan gene delivered with intramuscular instillations of adeno‐associated vectors. Hum. Gene Ther. 11, 777–790. , Bartlett, J. S. and Samulski, R. J. (1999). AlphaVbeta5 integrin: A co‐receptor for adeno‐associated virus type 2 infection [In Process Citation].
J. Virol. 76, 13015–13027. Qing, K. et al. (1999). Human fibroblast growth factor receptor 1 is a co‐ receptor for infection by adeno‐associated virus 2 [In Process Citation]. Nat. Med. 5, 71–77. Rabinowitz, J. E. et al. (2002). Cross‐packaging of a single adeno‐associated virus (AAV) type 2 vector genome into multiple AAV serotypes enables transduction with broad specificity. J. Virol. 76, 791–801. Richardson, W. D. and Westphal, H. (1981). A cascade of adenovirus early functions is required for expression of adeno‐associated virus.
Like other potential biopharmaceutical products, FDA, Center for Biologics Evaluation and Research (CBER) regulates the production, testing and use of AAV vectors in human clinical trials, in the US. Many regulatory documents are available to help guide the researcher. htm. Review of these documents prior to significant work towards production of vector for 36 ADENO-ASSOCIATED VIRUS VECTORS FOR GENE THERAPY clinical trial use is necessary to plan and implement an appropriate product development program.