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 treatment holds nice promise for the therapy of many genetic illnesses. as a way to in achieving this type of healing there are requisites. First, the affected gene needs to be cloned, its se­ quence decided and its rules thoroughly 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 sturdy shape, skill to introduce the gene into the specified phone variety, skill to specific the brought gene in an competently regulated demeanour for a longer time period, and an absence of toxicity for the recipient. additionally of outrage is the frequency of mobile transformation and, occasionally, the facility to introduce the gene into nondividing stem cells. Sev­ eral animal viruses were confirmed as strength vectors, yet none has confirmed to have the entire wanted houses defined above. for instance, retroviruses are tough to propagate in enough 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, hence, are in all probability insertional mutagens. also, genes brought by means of retroviral vectors are usually expressed for really brief classes of time. A moment virus used as a vector in version structures has been adenovirus (Ad).

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Transient expression assays indicate that Rep may also function posttranscriptionally to repress gene expression, as indicated by increased levels of transcripts and a concomitant decrease of reporter gene product activity (unpublished observations; TREMPE and CARTER 1988). Strategies for influencing cellular macromolecular synthesis employed by viruses include affecting message stability or transport (PILDER et al. 1986) or translational initiation (KAlZE et al. 1986; KRAUSSLICH et al. 1987). AAVSl CpG Integration •1 kb• Pl 5' CGGGCGGGTGGTGGCGGC GGTTGG GGCTCGGC GCTCGCTCGCTCGCTG GGCGGGCGGGCGGT 111111111111111111111111 11111111 1111111111111111 11111111111111 3' GCCCGCCCACCACCGCCG CCAACC CCGAGCCG CGAGCGAGCGAGCGAC CCGCCCGCCCGCCA I A A A C- G I I Rep Binding G- C I TRS G- C Motif G- C C C - G C' C- G AAV ITR C- G 0' I A G- C 5' AGGAACCCCTAGGATGG AGTTCC CCACTCCCTCTCT GCGCGCTCGCTCGCTC ACTGAGGCC - G T 11111111111111111111111 1111111111111 1111111111111111 111111111 3' TCCTTGGGGATCCTACC TCAAGG GGTGAGGGAGAGA CGCGCGAGCGAGCGAG TGACTCCGG - C C- G A' C- G o C- G G- C B C - G B' T- A G- C G- C T T T I I I Fig.

Packaging via Plasmid Transfections . . . . . . . . . . Adeno-associated Virus Protein-Expressing Cell Lines 39 39 41 42 4 Future Directions 45 5 Conclusions .................. References ........ 47 48 1 Introduction The promise and hopes that gene therapy vectors derived from adeno-associated virus (AAV) will make the leap from the laboratory bench to the clinic bedside are thoroughly described in this volume of Current Topics of MicrobiologV and Immunology. As more investigators enter this rapidly growing field, AAV's ability to deliver genes of therapeutic potential will be explored for many acquired and hereditary diseases.

In the THOMSON et al. (1994) report, there was no quantitation of the efficiency with which HHV-6 supports MV replication. This is a crucial question that might determine the fate of such an approach for packaging MV vectors. If HHV-6 is an inefficient helper of wild-type MV, its usefulness for producing high titer vector preparation would be suspect. The use of a herpes virus as a helper might be preferred over Ad because its large size and susceptibility to detergents should make it easier to separate from rMV.

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