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
Chaperoniterapia has shown promise in various conditions, including certain types of cystic fibrosis, lysosomal storage diseases,