Molecular chaperone therapy

Molecular chaperone therapy is one of the latest pharmacological approaches to lysosomal storage diseases. It fixes defective protein as an alternative to Stop codon suppression treatment. These chaperones are minute molecules that can enter the central nervous system ( via Blood Brain Barrier). Once in the CNS, they attach to the enzyme (inactive form) and fix it so that it takes the correct functional shape. Limitation of the therapy is that it only works with certain mutations : Gaucher's disease, Fabry disease, Pompe disease and Late-onset Tay-Sachs disease. Lack of bio-marker and the potential to reduce the enzyme function if given incorrect dose are challenging aspects of current clinical trials.