Cited by 210RNA Interference and Gene DeliveryNanoparticle-Based Drug DeliveryBarrier Structure and Function Studies

Nanotechnology-based strategies for siRNA brain delivery for disease therapy

Zheng, Meng, Tao, Wei, Zou, Yan, Farokhzad, Omid C., Shi, Bingyang·2018·Trends in biotechnology

Abstract

Small interfering RNA (siRNA)-based gene silencing technology has demonstrated significant potential for treating brain-associated diseases. However, effective and safe systemic delivery of siRNA into the brain remains challenging because of biological barriers such as enzymatic degradation, short circulation lifetime, the blood-brain barrier (BBB), insufficient tissue penetration, cell endocytosis, and cytosolic transport. Nanotechnology offers intriguing potential for addressing these challenges in siRNA brain delivery in conjunction with chemical and biological modification strategies. In this review, we outline the challenges of systemic delivery of siRNA-based therapy for brain diseases, highlight recent advances in the development and engineering of siRNA nanomedicines for various brain diseases, and discuss our perspectives on this exciting research field for siRNA-based therapy towards more effective brain disease therapy.

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Crossref · 10.1016/j.tibtech.2018.01.006

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84 of 84 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]”

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Authors
Zheng, M., Tao, W., Zou, Y., Farokhzad, O. C., & Shi, B.
Affiliations
  • Henan University (CN)
  • Brigham and Women's Hospital (US)
Corresponding author
Shi, Bingyang
Venue
Trends in biotechnology
Publisher
Elsevier BV
Published
2018-05
Volume
36
Issue
5
Pages
562-575
ISSN
0167-7799 (ISSN-L)
OpenAlex
W2787845337
Cited by
210
References
84
Citations by year
2026: 15 · 2025: 31 · 2024: 34 · 2023: 22 · 2022: 32 · 2021: 23 · 2020: 24 · 2019: 23 · 2018: 5 · 2012: 1
FWCI
1.84
Type
journal article
Language
en
Open access
closed
Locations
  • Trends in biotechnology (publishedVersion)
  • PubMed (publishedVersion)
Funders
  • National Natural Science Foundation of China (31640027, 31600809, U1604177)
  • Program of China 1000-Talents Plan
Topics
  • Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
  • Physical Sciences › Materials Science › Biomaterials › Nanoparticle-Based Drug Delivery
  • Life Sciences › Neuroscience › Neurology › Barrier Structure and Function Studies
Keywords
siRNA delivery, brain drug delivery, blood-brain barrier, gene silencing, brain disorders, systemic drug delivery, chemical modification, biological modification, nanocarriers, endocytosis
MeSH terms
Animals, Brain Diseases, Disease Models, Animal, Humans, Drug Delivery Systems, RNA, Small Interfering, Nanostructures, Nanomedicine, Molecular Targeted Therapy

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