Analysis of the Therapeutic Strategies Used to Treat ALS Caused by TDP-43 Aggregation

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.