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What is the current status of clinical gene therapy trials for Chronic Granulomatous Disorder - Essay Example

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Chronic granulomatous disease is a ‘phagocytic disorder that is rare and genetically inherited, and it entails deterioration of the immune system, thus making the affected population vulnerable to various infections caused by bacteria and fungi (Kepenekli 2014: 1)…
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What is the current status of clinical gene therapy trials for Chronic Granulomatous Disorder
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Download file to see previous pages ests that the disease is present in ‘one in 250,000’ infants at birth, but the symptoms become evident only when the child reaches a few months of age (Assari 2006: 6). Only 21% of patients have survived the disease beyond the age of 7 previously, whereas the recent trends show improvement in the survival rates because of the ‘advancement in treatments’ (Assari 2006: 6). On the other hand, the disease can rarely occur in adults, and therefore, if someone has the problem of ‘recurrent infections’, it will be advisable to subject such individuals to an evaluation, irrespective of the age factor (Chung, Cyr & Ellis 2013: 2).
Gene therapy, which is the intervention in a disease or disorder through introducing genes into the affected cells to set right the impacts of ‘specific gene mutations’, is one of the methods used in the treatment of CGD (Dugal & Chaudhary 2012: 4). However, evidence suggests that in order for the treatment to be effective, the appropriate gene will have to be incorporated into the target cell ‘specifically, efficiently and stably’ (Dugal & Chaudhary 2012: 4). On the other hand, it is also necessary to use the most appropriate vector for the introduction of the gene or gene delivery into the human cell. Research studies have identified lentiviral vectors (LVs) to be one of the effective and the ‘most widely used’ vectors in gene therapy (Dugal & Chaudhary 2012: 4). A study conducted by Assari (2006: 6) on mice found that gene therapy, using recombinant retroviral vectors, is an effective treatment for the reconstruction of normal neutrophils and in building ‘resistance to pathogens’ such as Aspergillus. The author also contends that gene therapy, using retroviral vectors in two humans, has produced ‘encouraging results’ where both of them have attained remarkable levels of gene corrected cells and their clinical conditions show good improvement (Assari 2006: 6). Studies further substantiate the fact that gene ...Download file to see next pagesRead More
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