Domestic researchers have developed a new NANO material that can effectively inhibit the aggregation of the TDP-43 (Transactive response DNA-binding protein-43) protein, which is the cause of the intractable neurodegenerative disease amyotrophic lateral sclerosis (ALS).
Lee Young-ho, head researcher at the Korea Basic Science Institute (KBSI) Advanced Biomedicine Research Division, noted that through joint research with Professor Hong Young-bin from Dong-A University College of Medicine, Senior Researcher Nam Min-yeop from the Korea Brain Research Institute, and Professor Hong Byeong-hee from Seoul National University Department of Chemistry, they have demonstrated that graphene quantum dots (GQDs) effectively block the abnormal aggregation of the TDP-43 protein and protect nerve cells.
Amyotrophic lateral sclerosis is a fatal disease that causes degeneration of motor neurons, leading to muscle paralysis and respiratory failure. In most ALS patients, aggregation of the TDP-43 protein in the cytoplasm is observed. This is known to be a major cause of neuronal dysfunction and death. However, most existing treatments focus on neuroprotection or anti-inflammatory effects, with very few treatment methods that directly control protein aggregation.
To effectively regulate the aggregation of the TDP-43 protein, researchers focused on graphene quantum dots, a carbon-based NANO material with sizes in the nanometer scale (1 billionth of a meter). Graphene quantum dots have high biocompatibility and can cross the blood-brain barrier.
Through experiments, it was confirmed that graphene quantum dots interact directly with the TDP-43 protein, effectively blocking protein aggregation and reducing toxicity associated with the TDP-43 protein in nerve cells, thereby increasing cell survival rates. They also confirmed that graphene quantum dots protect nerve cells in mutation models of other causative proteins of ALS, such as FUS and C9orf72. This suggests that graphene quantum dots can inhibit protein aggregation commonly observed in other neurodegenerative diseases besides ALS.
Professor Hong Young-bin said, "Since we have validated the therapeutic effects of graphene quantum dots in various cellular and animal models of ALS, there is a high possibility that it will be applicable to the majority of ALS patients in the future," and added, "It is expected that it will take time for the treatment method to be commercialized as we must go through safety and efficacy verification through preclinical and clinical trials." Head researcher Lee Young-ho stated, "We will conduct further studies based on cellular and animal models and computer simulations to discover and develop substances that help overcome degenerative diseases, like graphene quantum dots."
The research results were published in the international academic journal of nanoscience, ACS Nano, on 4th and were selected as the cover paper.
References
ACS Nano (2025), DOI: https://doi.org/10.1021/acsnano.4c15283