Novel siRNA delivery strategy: a new "strand" in CNS translational medicine?
Open accessAbstract
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.
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Efficient delivery of siRNA into the brain is a remarkable obstacle to clinical applications.
“While RNAi holds great promise for treating genetic associated neurological disorders, efficient delivery of siRNA into the brain is still a remarkable obstacle to the clinical applications.[99–101]”
From RNA delivery biomaterials for the treatment of genetic and rare diseases (2019) · cites this paper as [101]
Huntington's disease is caused by heightened CAG repeats in the huntingtin (HTT) gene.
“The cause of HD is related to the heightened CAG repeats in the huntingtin (HTT) gene.[101, 106]”
From RNA delivery biomaterials for the treatment of genetic and rare diseases (2019) · cites this paper as [101]
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Zhao, W., Hou, X., Vick, O. G., & Dong, Y. (2019) · Biomaterials · doi:10.1016/j.biomaterials.2019.119291 · Cited by 82 · see citing works
- Authors
- Gherardini, L., Bardi, G., Gennaro, M., & Pizzorusso, T.
- Affiliations
- Istituto di Fisiologia Clinica (IT)
- Italian Institute of Technology (IT)
- Neuroscience Institute (IT)
- Corresponding author
- Pizzorusso, Tommaso
- Venue
- Cellular and Molecular Life Sciences
- Published
- 2013-03-19
- Volume
- 71
- Issue
- 1
- Pages
- 1-20
- ISSN
- 1420-682X (ISSN-L)
- 1420-9071
- PMC
- PMC11113879
- OpenAlex
- W2120167828
- Cited by
- 28
- References
- 200
- Citations by year
- 2026: 3 · 2025: 3 · 2023: 1 · 2022: 1 · 2020: 3 · 2019: 2 · 2018: 1 · 2017: 3 · 2016: 4 · 2015: 4 · 2014: 3
- FWCI
- 0.34
- Type
- journal article
- Language
- en
- Open access
- green
- www.ncbi.nlm.nih.gov
- Licence
- www.springer.com/tdm (tdm)
- Locations
- Cellular and Molecular Life Sciences (publishedVersion)
- PubMed Central (publishedVersion)
- PubMed (publishedVersion)
- Florence Research (University of Florence) (submittedVersion, other-oa)
- Scuola Normale Superiore di Pisa (submittedVersion, other-oa)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Life Sciences › Neuroscience › Neurology › Barrier Structure and Function Studies
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Keywords
- siRNA delivery, RNA interference, central nervous system disorders, gene therapy, viral vectors, non-viral vectors, brain drug delivery, preclinical models, translational medicine, blood-brain barrier
- MeSH terms
- Blood-Brain Barrier, Drug Carriers, Genetic Vectors, Humans, Nervous System Diseases, Viruses, RNA Interference, RNA, Small Interfering, Nanoparticles, Translational Research, Biomedical
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