Work

New biotechnological and nanomedicine strategies for treatment of lysosomal storage disorders

Year: 2010

Type: review

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.WIREs Nanomed Nanobiotechnol2010 2 189–204 This article is categorized under: Therapeutic Approaches and Drug Discovery > Emerging Technologies

Source: Wiley Interdisciplinary Reviews Nanomedicine and Nanobiotechnology

DOI: 10.1002/wnan.73

Author: Silvia Muro

Institutions: University of Maryland Biotechnology Institute, University of Maryland, College Park

Language: English

Topic: Lysosomal Storage Disorders Research

Subfield: Physiology

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: green

Funder: National Heart, Lung, and Blood Institute