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

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

Human gene remedy holds nice promise for the treatment of many genetic ailments. which will in attaining this sort of remedy there are requisites. First, the affected gene has to be cloned, its se­ quence decided and its legislation thoroughly characterised. moment, an appropriate vector for the supply of an exceptional replica of the affected gene needs to be to be had. 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 good shape, skill to introduce the gene into the specified cellphone sort, skill to precise the brought gene in an competently 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, from time to time, the power to introduce the gene into nondividing stem cells. Sev­ eral animal viruses were verified as capability vectors, yet none has confirmed to have all of the wanted homes 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 homes in a few hosts and since they combine at many websites within the genome and, therefore, are most likely insertional mutagens. also, genes brought by means of retroviral vectors are usually expressed for quite brief classes of time. A moment virus used as a vector in version structures has been adenovirus (Ad).

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

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26 3 Role of Rep in Targeted Integration ............... 29 4 Replicative Integration 29 References .. 31 1 Introduction Adeno-associated virus 2 (AAV) , a nonpathogenic human virus, can either integrate into host chromatin and remain latent or replicate following infection. The outcome depends on the cellular conditions. , during adenovirus coinfection), AAV gene expression is induced. The single-stranded AAV genome of 4680 nucleotides is organized into two open reading frames (ORFs) that encode structural capsid proteins (Cap) and nonstructural proteins (Rep) (Fig.

In an AAV and Ad coinfected cell, the copy number of replicative form AAV DNA approaches 1O%ellieading to a very high level of viral Rep and Cap proteins. Wild-type AAV genomes are always replicated and encapsidated more efficiently than rAAV genomes in the same cell (HERMoNATand MuzycZKA 1984; MCLAUGHLIN et al. 1988; Trempe and Carter, unpublished observation). Thus there may be other unidentified cis-acting elements in the genome that promote replication and packaging. Another limitation of the current technology is the need for cumbersome, labor-intensive purification of rAAV over multiple CsCI 2 gradients.

Using only the terminal repeat sequence as a promoter, FLOTIE et al. (1993) demonstrated efficient expression of a cat reporter gene or a truncated cystic fibrosis cDNA in several different cell lines. Although there are no canonical TATA boxes, the terminal repeats contain several Sp1 sites and strong homology to the consensus Inr site found in many genes (cited in FLOTIE et al. 1993). DNA sequence analysis and extensive mutagenesis of AAV has revealed that between the terminal repeat sequences are two large translation open reading frames (SRIVASTAVA et al.

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