Question

In your continued work at BigPharma, you are running additional experiments with previously developed, FDA approved drugs to see if they can be used off-label to assist with the treatment of neurodegenerative disorders. Originally, NeuroStop was developed to attenuate brain damage following stroke, and thus might be a good candidate drug for helping slow neurodegeneration. Presented here are data from your most recent experiments which used a variety of aged wildtype (control, WT) and knockout transgenic mice (labeled as the specific mutant), some of which were injected with either NeuroStop (drug) or vehicle (control solution). neuron density in motor cortex 600 500 400 300 vehicle 200 100 wildtype(wT) α-Synuclein mutant SOD-1 mutant axonal transport in upper motor neurons Before we even get started, what is the SOD-1 mutant a mouse model of? (2 points a) Parkinson's disease b) Alzheimer's disease c) ALS 3.5 Ok so, what is the α-synuclein mutant a mouse model of? (2 points) a) Parkinson's disease b) Alzheimer's disease c) ALS 1.5 SOD-1 mutant -vehicle 'NeuroStop Based on this data, do you think NeuroStop could be used off-label to help control Parkinson's disease? (2 point) a) yes b) no immunoreactivity in midbrain 30 25 20 Based on this data, how do you think NeuroStop might be working at the cellular level (aka what is its mechanism of action)? Make sure you explain why you think that using the ALL of these data and what you already know from lecture to support your view. There are likely a few possible correct answers to this question; just focus on ONE 15 10 Lewy bodies TH WT NeuroStop a-synucle in mutant Neurostop WT vehicle tential mechanism of action and do it justice in your response. ($5 sentences; 4 points) c-synucle in mutant vehicle

2.  Ok... so, what is the α-synuclein mutant a mouse model of? (2 points) a) Parkinson’s disease b) Alzheimer’s disease c) ALS

Answer 1

α-synuclein mutant mouse model has been used to understand the therapeutic targets and disease pathogenesis of Parkinson’s disease.

The application of knockout mice has improved the chances to study the expression of genes related to diseases. Since, α-synuclein is the only major protein found related to parkinsons disease, the incorporation of α-synuclein mutant mouse model is vital. α-synuclein mouse model is the most effective because α-synuclein is the only protein part recognized, that has straight effect on Parkinson’s disease progression and pathogenesis.

Parkinson’s disease is considered as the neurodegenerative disorder that affects one per cent of the population having an age over sixty. Identification of parkinsons disease is done by knowing the existence of lewy bodies (which are protein inclusions created by α-synuclein).

Though there exist various other models related to genetic anomalies, none of them are useful in Parkinson’s disease investigation.