Invention:
This invention is a molecule that targets the aberrant aggregation of a certain protein (TDP-43, associated with neurodegeneration) of neurons. The molecule binds to a specific site within the TDP-43 protein, and this helps prevent the unwanted aggregation of TDP-43 within neurons. The molecule has been shown to stop and even reverse neurodegeneration in experimental models.
Background:
Neurodegenerative diseases affect millions of people globally. Currently, most treatments for these diseases only include treating symptoms and not the root causes of the disease. In the past 10 years, a corollary component of neurodegenerative diseases has been identified as the RNA transcribing related protein TDP-43. This protein plays an important role in cellular functions but can become a problem when many of these proteins aggregate within a neuron, forming insoluble masses. Many types of neurodegeneration have been shown to have problematic TDP-43 proteinopathy involved including Amyotrophic lateral sclerosis (Lou Gehrig’s disease) Frontotemporal lobar degeneration (FTLD), Alzheimer’s disease and Parkinson’s disease.
Applications:
- Treatment of Neurodegenerative diseases such as
- Amyotrophic Lateral Sclerosis
- Frontotemporal Lobar Degeneration
- Alzheimer’s Disease
- Parkinson’s Disease
Advantages:
- Effective at stopping and even reversing neurodegeneration
- Targets disease causes rather than just alleviating symptoms
