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Viral vectors adeno-associated virus

Figure 14.2 Vectors used thus far in gene therapy trials. Others are mainly viral-based and include the use of pox, vaccinia and adeno-associated viruses, as well as herpes simplex virus. Data adapted from www.wiley. co.uk/genemed/clinical... Figure 14.2 Vectors used thus far in gene therapy trials. Others are mainly viral-based and include the use of pox, vaccinia and adeno-associated viruses, as well as herpes simplex virus. Data adapted from www.wiley. co.uk/genemed/clinical...
A number of additional viral types may also prove useful as vectors in the practice of gene therapy. Chief amongst these are the adenoviruses. Adeno-associated virus, the herpes virus, and a number of other viruses, are also being considered (Table 14.2). [Pg.428]

Additional viruses that may prove of some use as future viral vectors include adeno-associated virus and herpes virus. Adeno-associated virus is a very small, single-stranded DNA virus its genome consists of only two genes. It does not have the ability to replicate autonomously and can do so only in the presence of a co-infecting adenovirus (or other selected viruses). [Pg.429]

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. [Pg.65]

Viral Vectors. The direct administration of a viral vector (e.g., retrovirus, adenovirus, adeno-associated virus, herpes, vaccinia) to patients. [Pg.66]

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). [Pg.410]

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... [Pg.419]

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. [Pg.334]

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. [Pg.321]

The strategies just described are in principle also applicable to other viral vectors, and a number of studies describing such approaches have been published, mainly for adeno-associated viruses (AAV) [45-47] and retroviruses [48-50], It can be anticipated that this field will rapidly expand as the mechanisms of virus-host cell interactions are unraveled in detail. [Pg.270]

Sun, L., Li, J. and Xiao, X. (2000). Overcoming adeno-associated virus vector size limitation through viral DNA heterodimerization. Nat. Med. 6, 599-602. [Pg.17]

Wang, X. S. et al. (1998). Characterization of wild-type adeno-associated virus type 2-like particles generated during recombinant viral vector production and strategies for their elimination. J. Virol. 72, 5472-5480. [Pg.56]

Kapturczak, M. H., Flotte, T. and Atkinson, M. A. (2001). Adeno-associated virus (AAV) as a vehicle for therapeutic gene delivery Improvements in vector design and viral production enhance potential to prolong graft survival in pancreatic islet cell transplantation for the reversal of type 1 diabetes. Curr. Mol. Med. 1, 245-258. [Pg.152]

Haberman, R. et al. (2002). Therapeutic liabilities of in vivo viral vector tropism Adeno-associated virus vectors, NMDAR1 antisense, and focal seizure sensitivity. Mol. Ther. 6(4), 495-500. [Pg.216]

AAV adeno-associated virus is a non-pathogenic member of the parvovirus family that harbors a single-shanded DNA viral genome. Vectors derived from AAV can carry up to a 4.5-kb transgene and stably express a transgene in vivo months to several years. [Pg.766]

Adeno-associated virus (AAV) is a non-pathogenic member of the parvovirus family. Its single-stranded DNA viral genome requires co-infection with either adenovirus or HSV for its own replication/propagation. Wild-type AAV encodes two viral gene products, Rep and Cap, which function in rep-lication/integration and structural stability, respectively. Vectors... [Pg.711]

To produce stable and long-lasting knockdown, viral vectors have been developed to efficiently express siRNAs or short hairpin RNAs (shRNAs) in a wide variety of mammalian cells (Fig. 8). Expression vectors include standard plasmids as well as those made from adenoviruses, adeno-associated viruses (AAVs), onco-retroviruses, and lentiviruses. The viral expression systems have the added benefit of being able to deliver siRNA, shRNA, or miRNA expression cassettes efficiently into cell types that are otherwise difficult to transfect via common transfection or electroporation protocols.f ... [Pg.3151]

Lieber A, Steinwaerder DS, Carlson CA, Kay MA. 1999. Integrating adenovirus-adeno-associated virus hybrid vectors devoid of all viral genes. J. Virol. 73 9314-24... [Pg.438]


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See also in sourсe #XX -- [ Pg.241 , Pg.265 ]

See also in sourсe #XX -- [ Pg.153 , Pg.154 ]




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