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Gene Therapy Essay

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The second technique used in gene therapy is ligation, which is used to ‘glue’ the removed functioning ADA gene into the viral DNA so that recombinant circular DNA (cDNA) can be created. Viral DNA is obtained from a virus, such as an adenovirus (often used for gene therapy treatments) which has been ‘purified’ so that it does not cause harm (infect host with the virus) to the human body when introduced. The ADA gene is then spliced into the open vector (viral cDNA), using the enzyme ligase which glues the ADA gene into the viral DNA by bonding the sugar phosphate groups together along the backbone of the viral and human DNA. This means that recombinant cDNA has been formed using the viral cDNA from a virus such as an adenovirus and the…show more content…
The last technique used is transfection which is the process by which the recombinant cDNA is inserted into a adenovirus (chosen for this explanation as it is one of the commonly used viruses for this process in gene therapy). The adenoviruses used in this process have been modified so that it is replication defective, which means that certain sequences of its DNA that relate to the construction or production of new viruses have been removed. So that once in the human body the adenoviruses can only insert their DNA into cell nuclei and not produce more adenoviruses so that chance of infection is nullified.

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This is then introduced into the human body commonly by injection where it will target cells nuclei inserting its genetic material which contains the functioning ADA gene meaning that a large number of somatic cells will be able to produce the functioning ADA protein can be synthesised. It is worth noting that while gene therapy has been done successfully on patients with ADA deficiency before, the treatment explained above is not the only method and will not work for every patient. After the recombinant DNA has been amplified the desired amount, it must first be injected into adenoviruses which will be the viral vectors responsible for delivering the functioning gene into the human body. After the DNA has been inserted into the adenoviruses these are placed into a solution which is then
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