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

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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
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However the prevalence of such diseases is in areas where there are small community resulting in interbreeding, for example Huntington’s disease (HD) and Amyotrophic lateral sclerosis (ALS); are prime examples of such incidents. This class of diseases causes the apoptosis of neurons and surrounding cells. The outcome for the sufferers has often been death. However there is promising developments in gene therapy, including SiRNA therapy in the treatment of these diseases.

Small Interfering RNA (SiRNA) was discovered by Andrew Fire and Craig Mello, receiving the Nobel Prize in 2006 for their work. (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
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