The size of the genome is 35kb, which is surrounded by the icosahedral capsid made up of 12 different proteins. The gag gene encodes for the viral core protein. The overexpression of oncogenes can be lessened by using this method. with these terms and conditions. Although, it is as effective as electroporation. Challenges & future aspect of gene therapy: What is Gene Therapy? Gene therapy is a strategy used to treat disease by correcting defective genes or modifying how genes they are expressed. However, the method is known since 1960. The viral vectors used in gene therapy can infect the host cells and produce a strong immune response against it. The viral gene expression can be divided into early and late phases, for understanding this we have to understand the structure of it. The liquid solution of DNA is encapsulated with the liposome lipid bilayer which helps in penetrating it. The treatment was developed by Carl H. June. The rep gene encodes for the protein that helps the AAV to integrate into the host genome, especially on chromosome 9. Conclusively, the in vivo gene therapy is not a good option for treating inherited diseases for now. The DNA can not directly be inserted into the host cell, so we need to create pores on the cell surface. However, direct injection of a transgene into the embryo is preferred more. The opinions expressed here are the views of the writer and do not necessarily reflect the views and opinions of News Medical. Cystic fibrosis and Duchenne muscular dystrophy can not be treated using it. Majorly, it infects the cells of upper respiratory airways with a long-lasting expression of more than 6 months. The env gene removed from the retrovirus and used to infect the target cell. The mechanism of germline gene therapy is shown in the figure below. After several years of it, in 1971, Carl R. Merril experimented on Human Fibroblast cells and concluded that DNA could be inserted in the human genome for fixing the mutant gene.eval(ez_write_tag([[336,280],'geneticeducation_co_in-medrectangle-4','ezslot_7',111,'0','0'])); Later on, in 1972 Theodore Friedman and coworkers successfully corrected the Lesch-Nyhan single gene disorder using gene therapy trials at the National Institute of Health. Under the strict sterile condition, altered cells are grown. In this way, the therapy alters the genome of future generations to come. And therefore it is not a good option for DMD and CF, we had already discussed it in the ex vivo gene therapy.eval(ez_write_tag([[250,250],'geneticeducation_co_in-netboard-1','ezslot_27',124,'0','0']));eval(ez_write_tag([[250,250],'geneticeducation_co_in-netboard-1','ezslot_28',124,'0','1'])); It can only insert DNA up to ~8kb, therefore the size of DNA transfer is limited. Polyethyleneglycol (PEG) is one of the best nanoparticle used in gene therapy. The transposons are mobile genetic elements that can move from one location to another into the genome. https://www.news-medical.net/health/Gene-Therapy-Types.aspx. Type of gene therapy. Although theoretically this could counteract hereditary disease, jurisdictions in various countries such as Switzerland, Australia and Germany prohibit the use of germline therapy due to fears over unknown risks and long-term effects in future generations. After inserting into the host cell, the pol gene encodes for the reverse transcriptase and ensured the integration of target DNA into the host genome. The magnetofection method is more suitable for ex vivo applications. Furthermore, aggressive strategies for the DMD and other related disorders suggest that there will be a lot of progress observed in the coming years. Robertson, Sally. 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