In the gene augmentation therapy, the mutant (LOF gene) is replaced by the fully functional wild type gene that translated a wild type protein again. The concept of gene therapy was originated in the 60s by Rogers and Pfuderer. Certain gene therapy techniques insert genes into cancer cells that allow conversion of an inactive drug called a pro-drug into the active form. Rapid integration of transgene is not possible, therefore, we don’t know when will the transgene be expressed. The transgene can be inserted into the muscle cell for the production of proteins such as insulin and thrombotic factors. The expression of the adenovirus divided into two phases the early infection phase and the late phase. The retrovirus is an RNA virus, the genome of it is made up of the RNA (not DNA). 2014: Adeno-associated virus-mediated gene therapy is used for the treatment of choroideremia disease, a rare form of the x-linked recessive disorder of vision loss. The frequently used vectors for ex vivo gene therapy are retrovirus and AAV. Exogenous gene is inserted in the defective cell lines using viral vectors. “. This is the reason, SCGT success rate is more for blood born disorders such as thalassemia, sickle cell anemia, and hemophilia. By the mechanism of reverse transcription, the RNA forms DNA (more specifically called cDNA), and the DNA is integrated into the genome of an organism. The retrovirus infects all types of body cells, consequently, it is not accurately target-specific. Also, instead of highly pressurized gas, electrical current or electrical discharge method can also be used. The effect of in vivo gene therapy is restricted to some areas. The vectors are divided into two broader categories: Some of the common types of virus used in the gene therapy are listed here. The method is also known as ballistic DNA transfer, micro-projectile gene transfer or DNA-coated particle bombardment. The last plasmid contains a gene of interest with the promoter and the Ψ sequence so that the mature virion particles are formed but with the GOI (not gag, pol, and env). Polyethyleneimine (PEI) is a gene delivery vector that helps in escaping GOI from the endosome. It is majorly used in the delivery of siRNA. Treating cancer using transgene is another utilization of gene therapy in which the expression of an oncogene is suppressed by using a transgene. The American Lung Association say to wear masks to stop the spread of COVID-19; Here’s why, The Prospects of Semaglutide for Treatment of Type 2 Diabetes Patients, Study pinpoints specific areas of the brain where serotonin promotes patience, Researchers study the role of viral intramembrane interactions in controlling programmed cell death, New system allows for individual supply of disinfected air in enclosed spaces for daily use, Mine ponds increase risk of mercury exposure for indigenous communities and wildlife, MARA project aims to use new DNA based nanotechnology to fight bacteria, UC Davis uses microneedles to deliver gene therapy for retinal diseases. These are described in more detail below. The gag gene encodes for the viral core protein. Some gene therapy techniques are designed to prevent this inhibition of apoptosis to ensure that the cancer cells do in fact die rather than survive. This was very informative and helpful for my grade 12 project, Thank you for this. As we are playing with live embryo here, some scientists believe that it is unethical. Overexpression of a gene causes some life-threatening disease conditions like cancer. The summarised history of the gene therapy is given below. In cancer cells, apoptosis is stopped and the cells divide to form new cells that also contain the damage DNA. Consequently, the change made in the DNA of these cells can pass it to the next generations.
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