Gene therapy vector

Lentiviruses are promising vectors for gene delivery in vivo with many in precli-nical development for gene therapy (115). The mechanism of nuclear targeting 

Because of the immunogenicity and biosafety issues associated with viral vectors, a significant number of researchers in the gene therapy community believe that nonviral gene delivery vehicles will be the vectors of choice in clinical gene therapy applications. Nonviral vectors are less immunogenic, can be 

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DNA Vaccination and Genetic Vaccination Gene-therapy Vectors... 

The development of AAV-based gene-therapy vectors is presently focused on the analysis of the properties of the different AAV serotypes, especially the host range of the virion shells of the different serotypes. By packaging... 

Gene-therapy Vectors. Table 1 Gene therapy - clinical trials... 

To circumvent this problem, vectors that are based on lentiviruses have been developed. In contrast to prototypic retroviruses, lentiviruses do not require cell division for integration. Gene-therapy vectors have been developed from a broad spectrum of lentiviruses including human immunodeficiency vims (HIV), simian and feline immunodeficiency vims as well as visna/maedi vims. The most widely used lentiviral vector system is based on HIV-1. These vectors can efficiently transduce a broad spectrum of dividing and nondividing cells including neurons, hepatocytes, muscle cells, and hematopoietic stem cells [1,2]. 

Finally, lentiviral vectors have been shown to transduce human embryonic stem cells. Therefore, this type of gene-therapy vector might also be used in stem cell-based therapies. 

Gene-therapy Vectors. Table 2 List of most common therapeutic genes used in gene-therapy trials (viral and non viral vectors)... 

Gene Expression Analysis Gene Gun Gene Products Gene Promoter Gene Therapy Gene-therapy Vectors Gene Transfer... 

Fenske DB, MacLachlan I, Cullis PR. Stabilized plasmid-lipid particles a systemic gene therapy vector. In Phillips MI, ed. Methods in Enzymology Gene Therapy Methods. Vol. 346. San Diego, CA, U.S.A. Academic Press Inc., 2002 36-71. 

Barry MA, Dower WJ, Johnston SA. Toward cell-targeting gene therapy vectors selection of cell-binding peptides from random peptide-presenting phage libraries. Nat Med 1996 2(3) 299-305. 

The system used to deliver the desired gene into the cells, called the gene therapy vector, may be a virus, a plasmid, or even just the naked DNA. The choice of vector is based on the difficulty of getting the gene into the cell, the amount of DNA the vector can carry, the size of the gene sequence needed to provide the correct protein, and the effects the vector may have on the body. Each type of vector has advantages and disadvantages. 

Viruses are very important as gene therapy vectors. Over the millennia of their existence, they have evolved to be very good at... 

Figure 6.1 An RNA viral gene therapy vector, engineered to carry the genetic information for a protein product, is incubated with blood or bone marrow cells removed from a patient. The foreign DNA sequence integrates into the cell s genetic material and when the cells are returned to the patient, they direct the production of the protein product. RNA gene therapy vectors are engineered to be unable to direct the production of new virus particles.

All of the following are desirable characteristics in the design of a gene therapy vector EXCEPT... 

Figures are not essential in a patent specification, but it is often the simplest way of including information such as a nucleotide sequence, or details of a purification process for a protein, or details of a new gene therapy vector.

Xiao W, Chirmule N, Berta SC, McCullough B, et al. 1999. Gene therapy vectors based on adeno-associated virus type 1. J Virol. 73 3994-4003. 

Most of today s approved biotechnology products are produced in bacteria, yeast, or mammalian cells. Newer sources currently used to manufacture clinical trial materials include insect cells, transgenic animals, and gene therapy vectors. Other potential sources include transgenic plants and nonviral delivery systems... 

J. G. Hay, N. Shapiro, H. Sauthoff, S. Heitner, W. Phupakdi, and W. N. Rom, Targeting the replication of adenoviral gene therapy vectors to lung cancer cells the importance of the adenoviral Elb-55kD gene, Hum. Gene Ther. 10 579 (1999). 

Ferrari, F. K., Xiao, X., McCarty, D. and Samulski, R. J. (1997). New developments in the generation of Ad-free, high-titer rAAV gene therapy vectors. Nat. Med. 3, 1295-1297. 

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