By K. I. Berns, C. Giraud (auth.), Professor Kenneth I. Berns M.D., Ph.D., Catherine Giraud Ph.D. (eds.)
Human gene treatment holds nice promise for the remedy of many genetic ailments. that allows you to in attaining the sort of remedy there are standards. First, the affected gene has to be cloned, its se quence made up our minds and its law properly characterised. moment, an appropriate vector for the supply of an excellent reproduction of the affected gene has to be on hand. For a vector to be of use numerous attributes are hugely fascinating: those contain skill to hold the intact gene (although this can be both the genomic or the cDNA shape) in a solid shape, skill to introduce the gene into the specified mobile variety, skill to precise the brought gene in an thoroughly regulated demeanour for a longer time period, and a scarcity of toxicity for the recipient. additionally of outrage is the frequency of mobile transformation and, often times, the facility to introduce the gene into nondividing stem cells. Sev eral animal viruses were established as strength vectors, yet none has confirmed to have all of the wanted houses defined above. for instance, retroviruses are tough to propagate in adequate titers, don't combine into nondividing cells, and are of outrage due to their oncogenic houses in a few hosts and since they combine at many websites within the genome and, therefore, are in all likelihood insertional mutagens. also, genes brought by way of retroviral vectors are often expressed for particularly brief classes of time. A moment virus used as a vector in version structures has been adenovirus (Ad).
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Additional info for Adeno-Associated Virus (AAV) Vectors in Gene Therapy
J Virol 63: 1023-1030 Yalkinoglu AO, Heilbronn R, Burkle A. Schlehofer JR, zur Hausen H (1988) DNA amplification of adenoassociated virus as a response to cellular genotoxic stress. Cancer Res 48: 3123-3129 Yang Q, Chen F, Trempe JP (1994) Characterization of cell lines that inducibly express the adenoassociated virus rep proteins. J Virol 68: 4847-4856 Zhou SZ, Broxmeyer HE, Cooper S, Harrington MA, Srivastava A (1993) Adeno-associated virus 2mediated gene transfer in murine hematopoieteic progenitor cells.
Various cell lines have been used successfully for this process including 293 cells (GRAHAM et al. 1977)' HeLa, and KB cells. The 293 cells may be the preferred cell line because of their high efficiency of transfection (ALWINE 1985). The basic design of an AAV vector packaging scheme is outlined in Fig. 2. The vector plasmid (prAAV) and the plasmid bearing the rep and cap genes (pAAV/Ad) are cotransfected onto Ad-infected tissue culture cells. In the presence of Ad, the Rep proteins rescue and amplify the recombinant AAV (rAAV) genome from the vector plasmid yielding a large pool of replicative form DNA.
1995). Rep cleaved duplex synthetic oligonucleotides corresponding to the P1 sequence predominantly at the predicted site within the TRS homolog (URCELAY et al. 1995). The cleavage efficiency of the synthetic P1 and the truncated ITR was similar (CHIORINI et al. 1994a). In addition, Rep formed a covalent complex with the P1 DNA substrate which is similar to established activity with either hairpinned (1M and MUZVCZKA 1990) or truncated ITR substrates (CHIORINI et al. 1994a). 4 Replicative Integration Under certain conditions, AAV DNA integrates into a specific region of human chromosome 19 (KOTIN et al.