TY - JOUR
T1 - Nanotechnology-Based Strategies for siRNA Brain Delivery for Disease Therapy
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.
UR - http://www.scopus.com/inward/record.url?scp=85041632936&partnerID=8YFLogxK
U2 - 10.1016/j.tibtech.2018.01.006
DO - 10.1016/j.tibtech.2018.01.006
M3 - Review article
C2 - 29422412
AN - SCOPUS:85041632936
SN - 0167-7799
VL - 36
SP - 562
EP - 575
JO - Trends in Biotechnology
JF - Trends in Biotechnology
IS - 5
ER -