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- W1016757764 abstract "Adeno-associated virus (AAV)-based vectors can achieve stable gene transfer without noticeable vector-related toxicities. However, the AAV serotype 2-derived vector has a restricted tissue tropism and a low transduction efficiency, which hampers the further development of AAV as vector. The use of alternative serotypes demonstrated a significant improvement over AAV2. For serotypes 1, 4, 5 and 6, a pattern of very distinct tropisms was described leading to a serotype of choice depending on the target cell or tissue. In the search for more potent AAV vectors with enhanced performance profiles, polymerase chain reaction-based techniques were exploited to examine human and non-human primate tissues for the identification and isolation of endogenous AAVs. Over one hundred novel primate AAV proviral sequences were discovered. The molecular prevalence of endogenous AAVs was 18–19% in the tissues evaluated. Further analyses suggested that primate AAVs are clustered in clades according to their phylogenetic relatedness. The members of each clade share functional and serological similarities. Three novel clades, represented by serotypes AAV7, AAV8 and AAV9, yielded vectors that outperformed previously known AAV vector systems for several different target tissues. Although, to date, no studies have been reported that employ these novel primate vectors for the central nervous system-directed gene transfer, several other gene therapy applications for long-term phenotypic correction in mouse and canine disease models have already been accomplished. These newly identified AAV vectors represent a new generation of efficient vehicles for somatic gene transfer in different gene therapy applications. This review summarizes previously published work in this area." @default.
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- W1016757764 date "2006-01-01" @default.
- W1016757764 modified "2023-10-18" @default.
- W1016757764 title "Identification of Novel Adeno-Associated Virus Serotypes for Use as Vectors" @default.
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