|SS1|| Expanding Concept of Chaperone Therapy for Inherited Brain Diseases
Chaperone therapy is a new concept of molecular therapeutic approach, first developed for lysosomal diseases, utilizing small molecular competitive inhibitors of lysosomal enzymes. This concept has been gradually targeted to many diseases of other categories, utilizing various compounds not necessarily competitive inhibitors but also non-competitive inhibitors or endogenous protein chaperones (heat-shock proteins).
In this session we will discuss current trends of chaperone therapy targeting various types of neurological and non-neurological diseases caused by misfolded mutant proteins. This molecular approach will open a new therapeutic view for wide variety of diseases, genetic and non-genetic, and neurological and non-neurological, in the near future.
Yoshiyuki Suzuki, M.D., Ph.D., Tokyo Metropolitan Institute of Medical Science (Japan).