Titlebook: Gene Targeting Protocols; Eric B. Kmiec Book 2000 Springer Science+Business Media New York 2000

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Site-Directed Alteration of Genomic DNA by Small-Fragment Homologous Replacement,(.)is a technology that can be used to achieve a number of different ends, including:the introduction of specific mutations into vectors, development of transgenic animals, and gene therapy.Homologous replacement is versatile, in that sequences can be directly targeted and altered, inserted, or dele

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Mutation Correction by Homologous Recombination with an Adenovirus Vector,lian systems (.). Its wider application, especially to human gene therapy, is, however, hampered by its low level of efficiency. Only a very small fraction of the treated cells will acquire the designed change. The overall inefficiency may result from the rarity of precise homologous recombination a

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Site-Specific Targeting of DNA Plasmids to Chromosome 19 Using AAV , and , Sequences,d helper virus (i.e., adenovirus [Ad] or herpesvirus) to undergo productive infection. In the absence of a helper virus, AAV will integrate preferentially into the host chromosome (ch). Targeting of AAV to ch19.13.3 qter locus has been documented at a frequency of 70% or greater (.). Because of AAV’

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Adeno-Associated Virus Based Gene Therapy in Skeletal Muscle,ired diseases, with over 100 protocols approved by the National Institutes of Health since 1989. Skeletal muscle is an especially attractive target for gene therapy, because of its accessibility and capability to uptake, maintain, and express recombinant protein from plasmid DNA (.–.). Transduction

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EBV-Derived Episomes to Probe Chromatin Structure and Gene Expression in Human Cells,on initiation. Research in the past two decades has provided a wealth of information about the mechanisms of transcriptional control in human cells. The analysis of the promoter and enhancer DNA elements, and of the corresponding DNA-binding proteins, has provided an understanding of the basic mecha