Autophagy Against Alzheimer's Disease Essay

1653 Words4 Pages

Jessica Binder
Neuro 509 paper
(rough draft)
4/11/14

“Autophagy Against Alzheimer’s Disease”

The goal of the proposed research is to test the utility of autophagy against Alzheimer’s disease. Specifically by taking a genetic approach, using a master transcriptional regulator of autophagy, Transcription factor EB (TFEB). For hypothesis-testing purposes, the plan is to genetically accelerate autophagy in microglia, neurons, neuron-microglia, and in animal models of tauopathy. Thus we hypothesize that enhancing autophagy using genetic manipulation will block neuroinflammation, clear intracellular protein-aggregates (MAPT/Aβ), and improve cognitive function in mouse models of neurodegenerative tauopathy.
In 1906, Alois Alzheimer first described Alzheimer's disease (AD) using silver staining techniques to identify amyloid plaques and neurofibrillary tangles. In the brain, neurons connect and communicate at synapses, where tiny bursts of chemicals called neurotransmitters carry information from one cell to another. Alzheimer's disrupts this process, damaging the brain's communication network, and eventually destroys synapses leading to cell death. This phenomenon causes the brain to shrink as gaps develop in the temporal lobe and hippocampus, which are responsible for storing and retrieving new memories. AD is the most common cause of dementia and accounts for 50%-75% of all cases. The two major AD pathologies include amyloid-β peptide (Aβ)-derived extracellular senile plaques and neurofibrillary tangles (NFTs). NFTs consist of intraneuronal aggregates of hyperphosphorylated and misfolded tau that become extraneuronal (“ghost” tangles) when tangle-bearing neurons die. NFTs have a stereotypical spatiotemporal progression that corre...

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...neuroscience research. However, it is not yet clear whether this treatment can be easily applied to humans, besides primary cell culture. Most current experiments are genetically introducing the light-sensitive proteins into selected neurons of mice. This type of targeted neuronal gene therapy would be far more difficult in humans. The other obstacle is that the light activation in the mice required implantation of an optical fiber which directs the light into a specific area of the brain. Performing such an invasive procedure in patients could carry potential risks that would need to be carefully balanced with the risks and benefits. Hopefully, future improvements in gene therapy methods and light stimulation will be able to help overcome these obstacles and pave the way for a whole new class of optogenetics-based therapies in patients with neurological disorders.

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