Nanobody-based Protein Degraders for the Treatment of Neurodegenerative Diseases
Neurodegenerative diseases including Alzheimer's and Parkinson’s diseases, amyotrophic lateral sclerosis (ALS), prion diseases, and among others affect millions of people in the US. These diseases are relentless, often fatal, and affect fundamental functions – things like moving, breathing, speaking, and thinking.
Unfortunately, there is currently no adequate treatment or cure for these neurodegenerative diseases, despite many efforts to advance traditional pharmacology approaches. Neurodegenerative disorders are characterized by a progressive deterioration of brain function, with a significantly consequent decrease in the quality of life of patients and their families. Due to increasing life expectancy, the incidence of these diseases has increased over the years and there is a significant unmet medical need for new treatments for these devastating diseases.
Neurodegenerative diseases caused by the presence and accumulation of pathological proteins lead to progressive loss of structure and/or function of neurons, including the death of neurons. OSU researchers have developed a novel therapeutic Targeted Protein Degradation (TPD) system inspired by the PROteolysis TArgeting Chimeras (PROTAC) technology, called nanobody-based protein degraders (nano-degraders, NDs). These nano-degraders target the degradation of those pathological proteins in human neuronal models of the diseases with the potential to be effective therapies to treat human neurodegenerative conditions and have wide applicability to a range of other protein misfolding disorders.
Therapeutic targeting degradation of pathological proteins to treat a variety of neurodegenerative diseases
No cure is available for Alzheimer's and Parkinson’s diseases, amyotrophic lateral sclerosis (ALS), and other neurodegenerative diseases. The ability to induce protein degradation in a directed manner has the potential to be an effective treatment.