Cited by 28RNA Interference and Gene DeliveryBarrier Structure and Function StudiesVirus-based gene therapy research

Novel siRNA delivery strategy: a new "strand" in CNS translational medicine?

Open access
Gherardini, Lisa, Bardi, Giuseppe, Gennaro, Mariangela, Pizzorusso, Tommaso·2014·Cellular and Molecular Life Sciences

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.

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Registry

Crossref · 10.1007/s00018-013-1310-8

Cites

200 of 200 references verified0 citation errors · 0 not checkable

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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]”
  • 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]”

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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
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
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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