Titlebook: Gene Therapy for Neurological Disorders; Methods and Protocol Fredric P. Manfredsson Book 2016 Springer Science+Business Media New York 201

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Tissue-Specific Promoters in the CNSal targeting is first discussed in the context of vector tropism and appropriate delivery. Then, some of our own attempts to restrict expression of therapeutic factors to distinct brain cell populations are discussed, followed by a detailed description of the setscrews that are available for these e

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Small-Scale Recombinant Adeno-Associated Virus Purificationr and long-term expression in tissues including brain. In addition, rAAV has demonstrated an impressive safety profile in gene therapy trials. The emergence of rAAV serotypes with different cell tropisms and distribution properties has allowed scientists to tailor serotypes to specific experimental

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Controlling AAV Tropism in the Nervous System with Natural and Engineered Capsids an array of techniques for modification of the viral capsid. AAV capsid variants possess unique antigenic profiles and demonstrate distinct cellular tropisms driven by differences in receptor binding. AAV capsids can be chemically modified to alter tropism, can be produced as hybrid vectors that co

Monocle

Altering Tropism of rAAV by Directed Evolutionnvolves the generation of an initial library of high complexity followed by cycles of selection during which the library is progressively enriched for target-specific variants. Each selection cycle consists of the following: reconstitution of complete AAV genomes within plasmid molecules; production

RACE

Altering Entry Site Preference of Lentiviral Vectors into Neuronal Cells by Pseudotyping with Enveloudotyping of lentiviral vectors with different envelope glycoproteins not only confers the neurotropism to the vectors, but also alters the preference of sites of vector entry into neuronal cells. One major group of lentiviral vectors is a pseudotype with vesicular stomatitis virus glycoprotein (VSV

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Intraparenchymal Stereotaxic Delivery of rAAV and Special Considerations in Vector Handlingpowerful tool that provides the ability to manipulate gene expression in specific regions, or even specific cell types in the brain. Here, we describe the proper handling and stereotaxic delivery of recombinant adeno-associated virus to various neuroanatomical structures of the rodent brain.