The Use Of Animal Models And Sirna Technologies On Autosomal Dominantly Inherited Neurodegenerative Disease

3557 WordsOct 23, 201415 Pages
Andrew Wilson The suitability of the use of animal models and SiRNA Technologies in autosomal dominantly inherited neurodegenerative disease Dr P Martin Biomedical Science Abstract: The treatment of dominantly inherited neurodegenerative disease has been not feasible until recent discovers of gene therapy. Discovers such as short interfering RNA (siRNA) which cause gene silencing due to the molecule forming a complex with messenger RNA (mRNA), resulting in the degradation of mRNA through pathways in the cell. The siRNA properties shows the potential in the treatment of such diseases. However the advancements of such therapies require mouse models to carry trails, to allow the development of safe and reliable treatment when given to a…show more content…
(Kaur et al, 2012) Typically SiRNA are between 21 and 23 RNA nucleotides in length. SiRNA has the ability to cause the inactivity of a genes’ expression in somatic mammalian cells; has proven to be an exceptional tool for researchers for the control of disease-causing genes and theoretical treatments of inherited diseases in the future. However siRNA can only be used once the target mRNA sequence is known. (Sioud, 2004) However due to the Human Genome Project, a large portion of the target genes have been sequenced allowing SiRNA to become more practical. The siRNA molecule is the product of the RNA interference (RNAi) pathway. The starting molecule in the pathway is a long “triggering” double stranded RNA (dsRNA). The dsRNA is formed from either an RNAi molecule inserted into the cell or a siRNA which complexes with a specific strand of messenger RNA (mRNA). The formation of the dsRNA causes the pathway to start. The dsRNA must have 2 nucleotides which remain unpaired at the 3 prime terminal end. (Pushpataj et al, 2006) The dsRNA is converted to siRNA by the Dicer; an RnaseIII like enzyme. (Pushparaj et al, 2006) The dicer molecule contains a helicase domain suggesting that unwinding of the dsRNA must be necessary for the target recognition with base pairings with the enzyme substrate complex. (Pushparaj et al, 2006) The resulting siRNA from the dicer is incorporated in a roughly formed multi-subunit RNA silencing complex (RISC). The RISC
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