Neurodegenerative disease is an all-encompassing term for an array of conditions which principally affect the neurons of the human brain. These make up the nervous system, which also incorporates the spinal cord and, under normal circumstances, do not replace themselves or reproduce. Therefore, when they are damaged or die off, they are unable to be replaced. Neurodegenerative diseases are debilitating conditions that result in progressive degeneration and/or the death of nerve cells. This creates problems with movement, and mental functioning- known as Dementia. These diseases are said to be incurable (JPND Research, 2014).
One example of a dominantly inherited severe autosomal neurodegenerative disease is Huntington’s disease. It is an inherited neurodegenerative disorder, resulting from an expanded poly-glutamine repeat region in the Huntingtin (htt) gene, and many regions of the brain are affected by this (Dominguez; 2010). Huntington’s disease is caused by an abnormal polytglutamine (polyQ) expansion within a single gene- Huntintin (Htt) in this case- …show more content…
Antisense RNA molecules that target Htt mRNA have been known to impair the translation of the gene sequence into the protein in vitro. However, the process of designing an siRNA which would target the mutant CAG repeat does inevitably result in degradation of normal huntingtin mRNA, as well as many other genes that have CAG repeats. This means that the siRNAs that target CAG repeats are unable to differentiate between the wild-type and mutant Htt genes (Lo, Hughes; 2010). Yet, a study from 2007 showed that directly administrating siRNAs directed against Htt has some therapeutic potential. It discussed that siRNA therapy for autosomal dominantly inherited neurodegenerative disease shows potential because in theory, siRNA can be directed to the fundamental cause of HD, by silencing expression of the mutant protein (DiFiglia, et al;
One example of a dominantly inherited severe autosomal neurodegenerative disease is Huntington’s disease. It is an inherited neurodegenerative disorder, resulting from an expanded poly-glutamine repeat region in the Huntingtin (htt) gene, and many regions of the brain are affected by this (Dominguez; 2010). Huntington’s disease is caused by an abnormal polytglutamine (polyQ) expansion within a single gene- Huntintin (Htt) in this case- …show more content…
Antisense RNA molecules that target Htt mRNA have been known to impair the translation of the gene sequence into the protein in vitro. However, the process of designing an siRNA which would target the mutant CAG repeat does inevitably result in degradation of normal huntingtin mRNA, as well as many other genes that have CAG repeats. This means that the siRNAs that target CAG repeats are unable to differentiate between the wild-type and mutant Htt genes (Lo, Hughes; 2010). Yet, a study from 2007 showed that directly administrating siRNAs directed against Htt has some therapeutic potential. It discussed that siRNA therapy for autosomal dominantly inherited neurodegenerative disease shows potential because in theory, siRNA can be directed to the fundamental cause of HD, by silencing expression of the mutant protein (DiFiglia, et al;