3. Huntington\'s Disease (HD) is a rare and fatal neurodegenerative disorder tha
ID: 3506637 • Letter: 3
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3. Huntington's Disease (HD) is a rare and fatal neurodegenerative disorder that results in hyperkinesia. One of the issues that occurs as a result of Huntington's is deficits in axonal transport. For example, mutations in the huntingtin (Htt) gene result in the loss of anterograde transport of organelles such as mitochondria. Among their many functions, mitochondria are thought to sequester Ca2+ ions. You working with an animal model of HD. You are recording the responses of neurons receiving synaptic inputs from cells in normal tissue and HD tissue. You note that these responses occur 3-5x more often in the HD tissue than that of normal tissue. Propose a mechanism wherein the Htt mutation could produce this increase in the responses of the HD neurons.Explanation / Answer
Some research done on mice.this is a rare disease.this disease occur when a person inherit from one parent.a mutant copy of htt gene that contain extra repetation or a stutter. This lead to production of misfolded protein.that is toxic to brain neuron .that trigger a degenerative process.or mood swings that ultimately leads to death.
By research :use miss.and we inject millions of viral particle in 8months old mice body.we can htt gene productions in inner brain that controls motor gene.that trigger the early sign if the gene.we get result that Each particle of htt , like a Trojan horse, delivered to the neurons one of the two pieces of the CRISPR/Cas9 editing system: either a short guide RNA sequence to mark for removal the HTT gene’s CAG repeats or a scissor-like Cas9 enzyme to snip out the repeats. In this strategy, both the health and abnormal copies of the HTT gene were “knocked out,” resulting in the production of no HTT protein.
three weeks later, we found that the CRISPR/Cas9 gene editing had reversed the disease process in their mouse model. Neurons in the striatum had stopped making the HTT protein. What’s more, the toxic, abnormal HTT protein that had already clumped together in and around the neurons—and which likely would have would have killed them—had begun to clear to varying degrees in the mice. The same went for other protein abnormalities associated with the progression of Huntington’s disease.
Finally we can see that gene editing is a potential treatment for Huntington disease.
And we also saw that the synaptic response of htt gene is 3 to 4 times more than normal tissue.
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