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New biotechnological and nanomedicine strategies for treatment of lysosomal storage disorders
Author(s) -
Muro Silvia
Publication year - 2010
Publication title -
wiley interdisciplinary reviews: nanomedicine and nanobiotechnology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 2.175
H-Index - 72
eISSN - 1939-0041
pISSN - 1939-5116
DOI - 10.1002/wnan.73
Subject(s) - lysosomal storage disorders , substrate reduction therapy , nanomedicine , therapeutic modalities , drug discovery , medicine , enzyme replacement therapy , computational biology , nanotechnology , bioinformatics , biology , disease , pathology , materials science , nanoparticle , physical therapy
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. WIREs Nanomed Nanobiotechnol 2010 2 189–204 This article is categorized under: Therapeutic Approaches and Drug Discovery > Emerging Technologies