Enzyme replacement and enhancement therapies: lessons from lysosomal disorders.
The past decade has witnessed remarkable advances in our ability to treat inherited metabolic disorders, especially the lysosomal storage diseases, a group of more than 40 disorders, each of which is caused by the deficiency of a lysosomal enzyme or protein. During the past few years, both enzyme replacement and enhancement therapies have been developed to treat these disorders. This review discusses the successes and shortcomings of these therapeutic strategies, and the contributions that they have made to treating lysosomal storage diseases.
Desnick
Robert J
RJ
Department of Human Genetics, Mount Sinai School of Medicine at New York University, New York, New York 10029, USA. rjdesnick@mssm.edu
Schuchman
Edward H
EH
D000818:Animals; D002986:Clinical Trials as Topic; D004195:Disease Models, Animal; D057487:Enzyme Therapy; D004798:Enzymes / Q000096:biosynthesis; D000795:Fabry Disease / Q000188:drug therapy; D006801:Humans; D016464:Lysosomal Storage Diseases / Q000188:drug therapy*; D051379:Mice; D018832:Molecular Chaperones / Q000378:metabolism
D004798:Enzymes; D018832:Molecular Chaperones