Gene Therapy : A Method Of Manipulating Cells At The Molecular Level

2897 WordsOct 9, 201412 Pages
Gene therapy was first proposed as a method of manipulating cells at the molecular level through the transfer of defined genetic material into a genome for the ultimate purpose of preventing or altering rare genetic disease states. Viruses have the natural ability to deliver genetic material to cells, which makes them excellent vectors for gene delivery (Waehler, Russell, & Curiel, 2007). Lentivirus, Herpes Simplex Virus, Adenovirus and Adeno-Associated viruses (AAV) are among the most prominently used vectors for gene delivery to the therapeutic target. While, gene therapy is a promising treatment option for a range of diseases, there are still a number of unwarranted risks associated with the technique, where further study is…show more content…
While, most Parkinson cases are incidental, a proportion of cases appear hereditary and can be traced to specific genetic mutations. The neurologic abnormalities associated with Parkinson’s disease are known to result from degeneration of dopaminergic neurons and nigrostriatal pathways (Coune, Schneider, & Aebischer, 2012). Patients are typically treated with dopamine replacement therapy; however, long-term treatment leads to motor complications in addition to those that may already exist. Denyer & Douglas (2012) suggest that alternative gene therapy is capable of reversing the symptoms and progression of Parkinson’s disease by introduction of a viral vector adept in long term expression of transferrable genes and integration into chromosomal DNA. There are two main purported methods to potentiate symptomatic relief of Parkinson’s: by increasing basal ganglia dopamine levels through introduction of transgenes encoding for enzymes involved in dopamine production/ regulation or by modulating basal ganglia circuitry affected by PD (Denyer & Douglas, 2012). The primordial is supported by (Palfi et al., 2014) who showed a modified lentiviral vector was capable of transferring the genes required to encode for DOPA decarboxylase, tyrosine hydroxylase and GTP-cyclohydrolase 1 enzymes to the striatum in the brain via intrasatrial administration. Local and continuous
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