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Non-viral vectors for gene therapy [81] present certain advantages over viral methods, such as large scale production and low host immunogenicity. However, non-viral methods initially produced lower levels of transfection and gene expression, and thus lower therapeutic efficacy. Newer technologies offer promise of solving these problems, with ...
The use of non-viral vectors avoids many, but not all, of the defenses that cells employ against vectors. Plasmids, the circular DNAs shown in Fig. 1, are generally used for non-viral gene delivery. However, there are two major problems with most methods for delivering DNA to cellular chromosomes using plasmids, the most common form of non ...
How vectors work to transfer genetic material. Gene therapy utilizes the delivery of DNA into cells, which can be accomplished by several methods, summarized below. The two major classes of methods are those that use recombinant viruses (sometimes called biological nanoparticles or viral vectors) and those that use naked DNA or DNA complexes (non-viral methods).
The Sleeping Beauty transposon system (SBTS) is the first successful non-viral vector for incorporation of a gene cassette into a vertebrate genome. Up until the development of this system, the major problems with non-viral gene therapy have been the intracellular breakdown of the transgene due to it being recognized as Prokaryotes and the ...
Hydrodynamic Delivery (HD) is a method of DNA insertion in rodent models. Genes are delivered via injection into the bloodstream of the animal, and are expressed in the liver. This protocol is helpful to determine gene function, regulate gene expression, and develop pharmaceuticals in vivo. [1] W. Heston giving an intravenous injection to mice.
In gene therapy a gene that is intended for delivery is packaged into a replication-deficient viral particle to form a viral vector. [29] Viruses used for gene therapy to date include retrovirus, adenovirus, adeno-associated virus and herpes simplex virus. However, there are drawbacks to using viruses to deliver genes into cells.
Gene augmentation, [14] gene replacement, and novel transgene gene therapy [15] strategies for the potential medical management of osteoarthritis are under preliminary research to define pathological mechanisms and possible treatments for this chronic disease. While viral vector gene therapies predominate, both viral and non-viral vectors have ...
In 2004, it was proposed that non-viral episomes might be used in genetic therapy for long-term change in gene expression. [2] As of 1999, there were many known sequences of DNA (deoxyribonucleic acid) that allow a standard plasmid to become episomally retained. One example is the S/MAR sequence. [3]
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