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

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 therapy of many genetic illnesses. that allows you to in attaining the sort of healing there are requisites. First, the affected gene needs to be cloned, its se­ quence decided and its law correctly 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 solid shape, skill to introduce the gene into the specified cellphone style, skill to specific the brought gene in an properly regulated demeanour for a longer time period, and a scarcity of toxicity for the recipient. additionally of shock is the frequency of cellphone transformation and, sometimes, 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 tricky to propagate in adequate titers, don't combine into nondividing cells, and are of shock due to their oncogenic houses in a few hosts and since they combine at many websites within the genome and, hence, are probably insertional mutagens. also, genes brought by way of retroviral vectors are often expressed for quite 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

Sample text

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 filled circles are the locations of the transcription promoters.

1989). The minimal sequence required for AAV DNA replication and packaging has been refined to 145 bp (FLoTTE et al. 1993). A vector containing a truncated cystic fibrosis transmembrane conductance regulator cDNA and only 145 bp of AAV terminal repeat sequence at each end of the transcription cassette was efficiently encapsidated and used to transduce cells without selection. This observation increases the size of heterologous DNA that can be packaged into an AAV virion to approximately 4500 bases.

1989; KYOSTIO et al. 1994; LABOW et al. 1986; MCCARTY et al. 1994b; TREMPE and CARTER 1988). 1987). These cellular effects associated with Rep68 and 78 may result from direct interactions of Rep with cellular DNA. Although the secondary structure of the ITR was considered essential for Rep68 binding (ASHKTORAB and SRIVASTAVA 1989; 1M and MUZVCZKA 1990; SNYDER et al. 1993), recent work demonstrated that binding does not require a stem-loop structure and that Rep binds to a linear truncated form of the ITR with affinity similar to that of the wild-type hairpin ITR (CHIORINI et al.

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