Adeno-associated virus, for gene

Fisher, K. J., Gao, G. P., Weitzman, M. D., De Matteo, R., Burda, J. F. and Wilson, J. M. (1996). Transduction with recombinant adeno-associated virus for gene therapy is limited by leading-strand synthesis. J. Virol. 70, 520-532. 

Yan, Z., Zhang, Y., Duan, D. and Engelhardt, J. F. (2000). From the cover Trans-splicing vectors expand the utility of adeno-associated virus for gene therapy [see comments], Proc. Natl. Acad. Sci. USA 97, 6716-6721. 

Ponnazhagam S, Curiel DT, Shaw DR, Alvarez RD, et al. 2001. Adeno-associated virus for cancer gene therapy. Cane Res. 61 6313-6321. 

Clark, K. R., Voulgaropoulou, F., Fraley, D. M. and Johnson, P. R. (1995). Cell lines for the production of recombinant adeno-associated virus. Hum. Gene Ther. 6, 1329-1341. 

Adeno-associated virus mediated gene therapy for vascular retinopathies... 

Cordier, L, Gao, G. P., Hack, A. A., McNally, E. M., Wilson, J. M., Chirmule, N. and Sweeney, H. L. (2001). Muscle-specific promoters may be necessary for adeno-associated virus-mediated gene transfer in the treatment of muscular dystrophies. Hum. Gene Ther. 12, 205-215. 

Cordier, L. et al. Muscle-specific promotors maybe necessary for adeno-associated virus-mediated gene transfer in the treatment of muscular dystrophies. Hum. Gene Ther., 12,205, 2001. 

Collaco RF, Cao X, Trempe JP. A helper virus-free packaging system for reeombinant adeno-associated virus vectors. Gene 1999 238 397-405. 

Gene Therapies. The types of vectors that have been used or proposed for gene transduction include retrovirus, adenovirus, adeno-associated viruses, other viruses (e.g., herpes, vaccinia, etc.), and plasmid DNA. Methods for gene introduction include ex vivo replacement, drug delivery, marker studies, and others and in vivo, viral vectors, plasmid vectors, and vector producer cells. 

For loss-of-fundion mutations gene replacement therapy usng retroviruses, adenoviruses, adeno-associated viruses, or liposomes as vectors... 

BOX 15.2. ENGINEERING OF ADENO-ASSOCIATED VIRUS AS A GENE THERAPY FOR HEMOPHILIA... 

Recently another novel method has been described for factor VIII gene transfer in patients with hemophilia A that avoids the use of viral vectors. The transfer was safe and well tolerated, and provided a modicum of benefit [8], Up until now the most promising strategy for factor VIII gene transfer had relied on adeno-associated virus (AAV). 

Adeno-associated virus is the most innocuous of viral vectors. It is nonpathogenic and its genome can be engineered to only encode for the therapeutic gene. There... 

There is a wide variety of vectors used to deliver DNA or oligonucleotides into mammalian cells, either in vitro or in vivo. The most common vector systems are based on viral [retroviruses (9, 10), adeno-associated virus (AAV) (11), adenovirus (12, 13), herpes simplex virus (HSV) (14)] andnonviral [cationic liposomes (15,16), polymers and receptor-mediated polylysine-DNA] complexes (17). Other viral vectors that are currently under development are based on lentiviruses (18), human cytomegalovirus (CMV) (19), Epstein-Barr virus (EBV) (20), poxviruses (21), negative-strand RNA viruses (influenza virus), alphaviruses and herpesvirus saimiri (22). Also a hybrid adenoviral/retroviral vector has successfully been used for in vivo gene transduction (23). A simplified schematic representation of basic human gene therapy methods is described in Figure 13.1. 

Rabinowtz, J.E., Samulski, J. (1998). Adeno-associated virus expression systems for gene transfer. Curr. Opin. Biotechnol., 9, 470-475. 

Gao, G.P. (2002). Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy. Proc. Natl. Acad. Sci. U.S.A., 99, 11854—11859. 

Flotte TR, Carter BJ. 1997. In vivo gene therapy associated with adeno-associated virus vectors for cystic fibrosis. Adv. Pharmacol. 40 85-101. 

Loeb, J.E., Cordier, W.S., Harris, M.E., Weitzman, M.D. and Hope, T.J. (1999) Enhanced expression of transgenes from adeno-associated virus vectors with the woodchuck hepatitis virus posttranscriptional regulatory element implications for gene therapy. Hum. Gene Then, 10, 2295-2305. 

In general, adenoviral vectors are known to infect the target cells effectively, but it is noteworthy to keep in mind that safety always comes first because some adverse side effects could be critical to the health of patients. Other types of viral vectors, such as adeno-associated virus, also are used for gene therapy in many diseases, even though more studies on safety, as well as efficacy, still remain until successful human clinical use can be expected. As demonstrated by clinical reports, fusion of knowledge on the molecular biology of viral vectors and the diseases to be treated holds promises for the future of medicine. 

Laboratory Techniques in Biochemistry and Molecular Biology, Volume 31 Adeno-Associated Virus Vectors for Gene Therapy T. R. Flotte and K. I. Berns (Editors)... 

ADENO-ASSOCIATED VIRUS VECTORS FOR GENE THERAPY... 

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