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Recombinant AAV mediated genome engineering
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Recombinant AAV mediated genome engineering : ウィキペディア英語版
Recombinant AAV mediated genome engineering

Recombinant adeno-associated virus (rAAV) based genome engineering is a genome-editing platform centered on the use of rAAV vectors that enables insertion, deletion or substitiution of DNA sequences into the genomes of live mammalian cells. The technique builds on Capecchi and Smithies’ Nobel Prize–winning discovery that homologous recombination (HR), a natural hi-fidelity DNA repair mechanism, can be harnessed to perform precise genome alterations in mice. rAAV mediated genome-editing improves the efficiency of this technique to permit genome engineering in any pre-established and differentiated human cell line, which, in contrast to mouse ES cells, have low rates of HR.
The technique has been widely adopted for use in engineering human cell lines to generate isogenic human disease models. It has also been used to optimize bioproducer cell lines for the biomanufacturing of protein vaccines and therapeutics. In addition, due to the non-pathogenic nature of rAAV, it has emerged as a desirable vector for performing gene therapy in live patients.
==rAAV Vector==

The rAAV genome is built of single-stranded deoxyribonucleic acid (ssDNA), either positive- or negative-sensed, which is about 4.7 kilobase long. These single-stranded DNA viral vectors have high transduction rates and have a unique property of stimulating endogenous HR without causing double strand DNA breaks in the genome, which is typical of other homing endonuclease mediated genome editing methods.

File:Targeting-vectors-2005-onwards.jpg|Diagram of a typical rAAV vector (source: http://horizondiscovery.com/technology/gene-targeting/)


抄文引用元・出典: フリー百科事典『 ウィキペディア(Wikipedia)
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