New PDF release: Adeno-Associated Virus (AAV) Vectors in Gene Therapy

By K. I. Berns, C. Giraud (auth.), Professor Kenneth I. Berns M.D., Ph.D., Catherine Giraud Ph.D. (eds.)

ISBN-10: 3642802079

ISBN-13: 9783642802072

ISBN-10: 3642802095

ISBN-13: 9783642802096

Human gene treatment holds nice promise for the therapy of many genetic ailments. which will in achieving this sort of healing there are requisites. First, the affected gene has to be cloned, its se­ quence made up our minds and its legislation appropriately characterised. moment, an appropriate vector for the supply of an outstanding reproduction of the affected gene has to be on hand. For a vector to be of use a number of attributes are hugely fascinating: those comprise skill to hold the intact gene (although this can be both the genomic or the cDNA shape) in a sturdy shape, skill to introduce the gene into the specified telephone style, skill to precise the brought gene in an correctly regulated demeanour for a longer time period, and an absence of toxicity for the recipient. additionally of shock is the frequency of telephone transformation and, in certain cases, the facility to introduce the gene into nondividing stem cells. Sev­ eral animal viruses were demonstrated as strength vectors, yet none has confirmed to have all of the wanted homes defined above. for instance, retroviruses are tricky to propagate in adequate titers, don't combine into nondividing cells, and are of shock as a result of their oncogenic homes in a few hosts and since they combine at many websites within the genome and, therefore, are very likely insertional mutagens. also, genes brought by way of retroviral vectors are often expressed for rather brief classes of time. A moment virus used as a vector in version platforms has been adenovirus (Ad).

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Additional resources for Adeno-Associated Virus (AAV) Vectors in Gene Therapy

Example text

1986; BEATON et al. 1989; KYOSTIO et al. 1994). Rep78 and Rep68 also inhibit gene expression from a variety of heterologous transcription promoters (LABOW et al. P. n Pv pAAV/Ad Xb Xb []. I,\. Xb~. [] Fig. 1. Adeno-associated Virus (AAV) genome and plasm ids used in vector packaging. The AAV genome is indicated at the top of the figure. The locations of the replication (rep) and capsid (cap) genes are indicated by the brackets. The filled boxes on each end of the genome are the terminal repeat sequences.

The cell pellet is frozen and thawed three to four times and the lysate heated at 55°-60°C for 30-60 min to inactivate Ad. After a low speed centrifugation step to remove debris, the crude vector preparation is purified via one or more CsCI 2 equilibrium gradient centrifugations. 35 g/cm 3 (DELAMAZA and CARTER 1978). The heat inactivation step is sufficient to inactivate all of the Ad that may be produced and use of crude rAAV preparation in cell culture is a common practice. If the rAAV is to be used in animal experiments, vector purification should be performed via multiple CsCI 2 gradient centrifugations.

This mutant genome cannot normally form an infectious particle because of an unknown defect in the accumulation of single-stranded DNA. Therefore the Rep52 produced in the Ne06 cells is probably functional. We were also able to package a rAAV that contains only a cat reporter gene by cotransfecting a plasmid that expresses the AAV cap gene. This is further evidence that Ne06 and Ne040 cells supply all of the known Rep protein functions. An interesting feature of these cell lines is that, upon induction of Rep expression, cell proliferation slows dramatically.

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Adeno-Associated Virus (AAV) Vectors in Gene Therapy by K. I. Berns, C. Giraud (auth.), Professor Kenneth I. Berns M.D., Ph.D., Catherine Giraud Ph.D. (eds.)


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