2022 AMI Online Salon

Antisense Oligonucleotide Drugs for Amyotrophic Lateral Sclerosis

Project Detail

  • Entrant Name:  Shiyu Chen
  • Client: University of Illinois at Chicago
  • Copyright:  Shiyu Chen, 2022
  • Medium/software used: Visual Molecular Dynamics (VMD), 3dsMax, Zbrush, Adobe After Effects, Adobe Illustrator
  • Final presentation format: Videos on websites or social media
  • Primary Audience: Lay audience with biomedical science background

Project Description

The pathogenic GGGGCC (G4C2) repeat expansion on the C9orf72 gene, is the most common cause of amyotrophic lateral sclerosis (ALS). The aggregated toxic dipeptide repeat (DPR) proteins caused by the repeat expansion contributes to the motor neuron dysfunction in ALS. In the past few years, researches have found that a type of new drug, antisense oligonucleotides (ASO) may be helpful in reducing the toxic DPR proteins. This animation intends to explain the pathogenesis of C9orf72 related ALS, and introduce the mechanism of ASO drugs. The animation starts with an intracellular scene explaining what is G4C2 repeat expansion, and how is it causing motor neuron dysfunction. Then ASO drugs appears in the animation to explain how they address the pathological condition. An inset with a motor neuron extracellular scene was used accordingly to visually demonstrate the improvement of the pathological condition. The use of plain language and simple representative models are easy to understand for the lay audience with basic biomedical knowledge background.