Viral Vectors for Gene Therapy: Methods and Protocols
The promise of gene therapy can be realized only if workable vectors can be found to deliver therapeutic genes. In Viral Vectors for Gene Therapy: Methods and Protocols, leading researchers from academia and biotechnology describe proven molecular methods for the construction, development, and use of virus vectors for gene transfer and gene therapy. Offering detailed step-by-step instructions to ensure successful results, these experts detail the use of herpes viruses, adenoviruses, adeno-associated viruses, simple and complex retroviruses, including lentiviruses, and other virus systems for vector development and gene transfer. Additional chapters demonstrate the use of virus vectors in the brain and central nervous system. Each protocol includes a discussion of the principles involved, numerous charts and tables, ample references, and notes on possible problems, troubleshooting, and alternative procedures. Comprehensive and highly practical, Viral Vectors for Gene Therapy: Methods and Protocols provides not only researchers with the basic tools needed to design targeted gene delivery vectors, but also clinicians with an understanding of how to apply viral vectors to the treatment of genetic disorders.
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Acad adeno-associated virus adenovirus adenovirus vectors amplicon approx assay brain buffer capsid centrifuge clones concentration conﬂuent containing deleted dilution dish DMEM efﬁcient electroporation encoding ﬁlter ﬁnal ﬁrst FIV vectors ﬂuorescence gene delivery gene expression Gene Ther gene therapy gene transfer genetic genome helper virus herpes simplex virus human immunodeﬁciency virus Incubate infection integration lentiviral lentiviral vectors lysate medium Methods modiﬁed Molecular mouse mutant Natl neurons packaging cell line packaging signal particles pellet plaques plasmid plate Proc production promoter protein Protocols pseudotyped puriﬁcation rAAV receptor replication replication-competent reporter gene Resuspend retroviral vectors sequences simplex virus type solution speciﬁc splicing sterile Subheading supernatant target cells tissue culture titer transcription transduction transfection transgene transgene expression tube vector stocks Vectors for Gene viral genome Viral Vectors virion Virol viruses vitro vivo wild-type