New biotechnological and nanomedicine strategies for treatment of lysosomal storage disorders.
This review discusses the multiple bio- and nanotechnological strategies developed in the last few decades for treatment of a group of fatal genetic diseases termed lysosomal storage disorders. Some basic foundation on the biomedical causes and social and clinical relevance of these diseases is provided. Several treatment modalities, from those currently available to novel therapeutic approaches under development, are also discussed; these include gene and cell therapies, substrate reduction therapy, chemical chaperones, enzyme replacement therapy, multifunctional chimeras, targeting strategies, and drug carrier approaches.
Muro
Silvia
S
Center for Biosystems Research, University of Maryland Biotechnology Institute, College Park, MD 20742, USA. muro@umd.edu
D000818:Animals; D001709:Biotechnology / Q000379:methods*; D016503:Drug Delivery Systems; D056947:Enzyme Replacement Therapy; D015316:Genetic Therapy; D006801:Humans; D016464:Lysosomal Storage Diseases / Q000209:etiology / Q000628:therapy*; D050997:Nanomedicine / Q000379:methods*