Chaperoniterapia

Chaperoniterapia, also known as chaperone therapy or pharmacological chaperone therapy, is a treatment strategy aimed at correcting the cellular dysfunction caused by misfolded proteins. This approach focuses on using small molecules that bind to specific misfolded proteins, stabilizing them and facilitating their proper folding or trafficking within the cell. By assisting these proteins to achieve or maintain their correct three-dimensional structure, chaperoniterapia aims to restore their normal function.

This therapeutic strategy is particularly relevant for genetic disorders where a mutation leads to the production of a misfolded protein that may be unstable, non-functional, or targeted for degradation before it can perform its intended role. The chaperones can act as "pharmacological assistants," helping these proteins fold correctly, escape cellular quality control mechanisms that would otherwise eliminate them, and reach their designated cellular location. This can, in turn, alleviate the symptoms associated with the protein deficiency or dysfunction.

Chaperoniterapia has shown promise in various conditions, including certain types of cystic fibrosis, lysosomal storage diseases, and neurological disorders. Research in this area is ongoing, exploring new chaperone molecules and expanding their application to a wider range of diseases caused by protein misfolding. The development of chaperoniterapia represents a significant advancement in the field of targeted molecular therapies.