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- Title
Novel siRNA delivery strategy: a new 'strand' in CNS translational medicine?
- Authors
Gherardini, Lisa; Bardi, Giuseppe; Gennaro, Mariangela; Pizzorusso, Tommaso
- Abstract
RNA interference has been envisaged as a powerful tool for molecular and clinical investigation with a great potential for clinical applications. In recent years, increased understanding of cancer biology and stem cell biology has dramatically accelerated the development of technology for cell and gene therapy in these areas. This paper is a review of the most recent report of innovative use of siRNA to benefit several central nervous system diseases. Furthermore, a description is made of innovative strategies of delivery into the brain by means of viral and non-viral vectors with high potential for translation into clinical use. Problems are also highlighted that might hamper the transition from bench to bed, analyzing the lack of reliable preclinical models with predictive validity and the lack of effective delivery systems, which are able to overcome biological barriers and specifically reach the brain site of action.
- Subjects
SMALL interfering RNA; DRUG delivery systems; CENTRAL nervous system diseases; THERAPEUTICS; TRANSLATIONAL research; ONCOLOGY; CANCER treatment; STEM cell treatment
- Publication
Cellular & Molecular Life Sciences, 2014, Vol 71, Issue 1, p1
- ISSN
1420-682X
- Publication type
Article
- DOI
10.1007/s00018-013-1310-8