Nanotechnology-Based Strategies for siRNA Brain Delivery for Disease Therapy

Meng Zheng; Wei Tao; Yan Zou; Omid C. Farokhzad; Bingyang Shi

doi:10.1016/j.tibtech.2018.01.006

Nanotechnology-Based Strategies for siRNA Brain Delivery for Disease Therapy

Meng Zheng, Wei Tao, Yan Zou, Omid C. Farokhzad, Bingyang Shi^*

^*Corresponding author for this work

Research output: Contribution to journal › Review article › peer-review

191 Citations (Scopus)

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.

Original language	English
Pages (from-to)	562-575
Number of pages	14
Journal	Trends in Biotechnology
Volume	36
Issue number	5
DOIs	https://doi.org/10.1016/j.tibtech.2018.01.006
Publication status	Published - 1 May 2018
Externally published	Yes

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10.1016/j.tibtech.2018.01.006

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title = "Nanotechnology-Based Strategies for siRNA Brain Delivery for Disease Therapy",

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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AU - Zheng, Meng

AU - Tao, Wei

AU - Zou, Yan

AU - Farokhzad, Omid C.

AU - Shi, Bingyang

PY - 2018/5/1

Y1 - 2018/5/1

N2 - 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.

AB - 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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JO - Trends in Biotechnology

JF - Trends in Biotechnology

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Nanotechnology-Based Strategies for siRNA Brain Delivery for Disease Therapy

Abstract

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