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- W111489413 abstract "The use of adeno-associated virus (AAV) as a gene transfer vector has been steadily increasing over the past several years. A AV vectors have been particularly useful for applications where sustained gene expression is required. Prolonged in vivo expression following A AV treatment has been seen in the liver (1,2), brain (3,4), skeletal muscle (5,6), lung (7,8), and hematopoietic stem cells (9,10) of animal models. Therapeutic benefit from A AV treatment has been shown in a number of preclinical models of disease, including animal models of coagulopathies (11,12), lysosomal storage diseases (13,14), vision defects (15,16), and amino acid disorders (17). Clinical trials using A AV for the treatment of hemophilia B have begun, and early reports from these trials have been promising (18). In this introductory chapter to AAV, we will provide a brief overview of the molecular biology of this virus, an overview of methods of vector production, and a brief summary of the use of alternate AAV serotypes. The following chapters will then focus on specific methods and techniques for AAV transduction of the organs listed previously." @default.
- W111489413 created "2016-06-24" @default.
- W111489413 creator A5070033255 @default.
- W111489413 date "2004-02-17" @default.
- W111489413 modified "2023-09-23" @default.
- W111489413 title "Overview of Adeno-Associated Viral Vectors" @default.
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- W111489413 doi "https://doi.org/10.1385/1-59259-650-9:157" @default.
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