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Nanobiotechnology
Published in Firdos Alam Khan, Biotechnology Fundamentals, 2020
Nanoparticles have also been used in gene therapy. A pilot experiment involving gene therapy in human subjects has already been conducted for diseases like cystic fibrosis and muscular dystrophy. Three main types of gene-delivery systems have been described: (1) viral vectors; (2) nonviral vectors (particles and polymers); and (3) gene guns for direct injection of the genetic material into the target tissue. As viral vectors pose some serious problems, nonviral vectors are the gene-delivery system of choice. In this nonviral vector system, negatively charged plasmid DNA is condensed into nanoparticles that are 50–200 nm in size. The use of cationic lipids and cationic polymers gives a compact structure because of interaction between cationic material and anionic DNA. These compact structures also provide increased stability and uptake by the target cells. Some of the nonviral vectors for gene therapy based on nanoparticles are listed in Table 10.2. The targets for gene therapy using nanoparticles include liver hepatocytes, endothelial cells, the spleen, and lymph nodes, where some success has already been achieved.
Carbon Nanomaterials for Biomedical Applications
Published in Kun Zhou, Carbon Nanomaterials, 2020
Hong Wu, Qianli Huang, Yanni Tan
Gene delivery is to import a gene into cells and then let these cells produce the therapeutic materials they need. Actually, gene therapy technology has two main sorts: viral gene delivery and nonviral gene delivery. For viral gene delivery, the foreign gene is attached to the virus body, which has been modified by removing the harmful parts and then introduced into the cell to realize gene delivery. All viruses can be developed as a tool for gene transfer through a series of treatments. However, because of the difference in the life cycle and molecular pathology of different viruses, four kinds of virus carriers are mainly used: retrovirus vector (including lentivirus vector), adenovirus vector, adeno-associated virus carrier, and herpes simplex virus carrier. Nonviral transmission is carried through DNA or attached to lipids or polymers and then inserted into different cells. Nonviral vectors are favored by many researchers and clinicians because of their advantages such as no infectivity, no restriction on carrier capacity, and mass product ability. There are four kinds of nonviral DNA transfer methods: bare DNA, liposomes, polymers, and molecular conjugates. In the way of gene delivery, carbon nanomaterials, especially CNTs and GR, are extensively researched as well as applied for gene-delivery applications.
Nano-biotechnology
Published in Firdos Alam Khan, Biotechnology Fundamentals, 2018
Nanoparticles have also found use in the field of nanotechnology. A pilot experiment involving gene therapy in human subjects has already been conducted for diseases like cystic fibrosis and muscular dystrophy. Three main types of gene-delivery systems have been described: (1) viral vectors; (2) nonviral vectors (particles and polymers); and (3) gene guns for direct injection of the genetic material into the target tissue. Since viral vectors pose some serious problems, nonviral vectors are the gene-delivery system of choice. In this nonviral vector system, negatively charged plasmid DNA is condensed into nanoparticles that are 50–200 nm in size. The use of cationic lipids and cationic polymers gives a compact structure due to interaction between cationic material and anionic DNA. These compact structures also provide increased stability and uptake by the target cells. Some of the nonviral vectors for gene therapy based on nanoparticles are listed in Table 10.2. The targets for gene therapy using nanoparticles include liver hepatocytes, endothelial cells, the spleen, and lymph nodes, where some success has already been achieved.
Gene doping: Present and future
Published in European Journal of Sport Science, 2020
Rebeca Araujo Cantelmo, Alessandra Pereira da Silva, Celso Teixeira Mendes-Junior, Daniel Junqueira Dorta
There are some molecular tools, like micro-seeding gene therapy, cationic liposomes, macromolecular conjugate, and gene-activated matrixes, that can be used to deliver a modified version of a gene, but the use of viral vectors stood out for a long time as the most effective means for gene delivery. Retroviruses and adeno-associated viruses have been the most successful vectors in the clinical setting (Dunbar, High, Joung, Ozawa, & Sadelain, 2018; Karthikeyan & Pradeep, 2006). These viruses are an excellent strategy to modify cells: they are programmed to transfect the cells by inserting their genetic material inside the cell and integrating it into the host genome, and they multiply by using resources from the host cells. Moreover, they have low immunogenicity, are a stable support for gene expression, and can be tissue-specific. Nevertheless, these viruses are attenuated, and their replicating activity is controlled during gene therapy (Beiter et al., 2011; van der Gronde, de Hon, Haisma, & Pieters, 2013).