Osmolytes as a Promising Therapeutic Strategy for Protein Aggregation Diseases

Protein aggregation and misfolding are central factors in the pathogenesis of various disorders, emphasizing their significant contribution to the development of neurodegenerative conditions. The accumulation of aggregated or misfolded proteins disrupts cellular function and leads to pathological conditions. Osmolytes are small organic compounds with low molecular weight and are crucial in cellular protection during stress or disease states. These molecules accumulate in tissues in response to such conditions, stabilizing aggregated proteins and preventing their misfolding. Osmolytes serve as adequate chemical chaperones, actively enhancing the stability of adequately folded functional proteins. They exert their influence by redirecting the equilibrium of protein folding away from the pathways that lead to aggregation and degradation. This chapter explores the significance of different classes of osmolytes in modulating protein aggregation, including free amino acids, sugars, polyhydric alcohols, derivatives, urea, and methylamine compounds. Osmolytes can prevent protein aggregation, interact rapidly with surrounding water molecules, and hinder protein misfolding by providing stability to the protein structure. Leveraging the properties of osmolytes opens up possibilities for several industrial applications and therapeutic interventions for various neurodegenerative diseases. By shedding light on the impact and mechanisms of action of osmolytes on protein aggregation, this chapter provides valuable insights into their role in different industries and their potential to develop novel treatments for various disorders.