Analysis of the Therapeutic Strategies Used to Treat ALS Caused by TDP-43 Aggregation
Project by Polygence alum Sonya
The outcome of this project was publication. It has currently been accepted for pre-print, and it is in the process of being peer-reviewed for publication in a journal.
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Amyotrophic lateral sclerosis (ALS), a fatal neurodegenerative disorder that impairs motor functions, affects 1 in 50,000 people in the world, and almost 90% of patients diagnosed do not have any family history of the disease. ALS is a debilitating disease due to the limited effectiveness of treatments for most patients. However, researchers were able to identify one protein that malfunctions in almost 97% of patients: TDP-43. TDP-43 is a protein that regulates the process of transcription, and it is known to aggregate in the neurons of patients with ALS. Many researchers have decided to focus their therapeutic strategies on the protein aggregation using genetic therapies on small molecules. This study focuses on analyzing the benefits and limitations of the therapeutic strategies used to treat ALS caused by TDP-43 aggregation and identifying which method holds the most promise to target this proteinopathy.
PhD Doctor of Philosophy candidate
neuroscience, stem cells, gene editing, CRISPR-Cas9
Review of mentor
Gokul was helpful in guiding me through the process of deriving my research question and creating my outline. He was also incredibly helpful with the writing portion of the research paper and answered all my questions.
I was able to investigate the research question that I determined, and I was able to determine a solution with the help of my mentor. I was also able to analyze all the previous tried methods, and altogether, this experience helped me become more knowledgeable in the field that I want to pursue.