New biotechnological and nanomedicine strategies for treatment of lysosomal storage disorders

Wiley Interdiscip Rev Nanomed Nanobiotechnol. Mar-Apr 2010;2(2):189-204. doi: 10.1002/wnan.73.


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.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • Biotechnology / methods*
  • Drug Delivery Systems
  • Enzyme Replacement Therapy
  • Genetic Therapy
  • Humans
  • Lysosomal Storage Diseases / etiology
  • Lysosomal Storage Diseases / therapy*
  • Nanomedicine / methods*