New biotechnological and nanomedicine strategies for treatment of lysosomal storage disorders.

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TitleNew biotechnological and nanomedicine strategies for treatment of lysosomal storage disorders.
Publication TypeJournal Article
Year of Publication2010
AuthorsMuro, S
JournalWiley Interdiscip Rev Nanomed Nanobiotechnol
Volume2
Issue2
Pagination189-204
Date Published2010 Mar-Apr
ISSN1939-0041
KeywordsAnimals, Biotechnology, Drug Delivery Systems, Enzyme Replacement Therapy, Genetic Therapy, Humans, Lysosomal Storage Diseases, Nanomedicine
Abstract

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.

DOI10.1002/wnan.73
Alternate JournalWiley Interdiscip Rev Nanomed Nanobiotechnol
PubMed ID20112244
Grant ListR01 HL098416-03 / HL / NHLBI NIH HHS / United States