Advances in cell-type specific delivery of RNAi-based therapeutics.
RNAi-based gene silencing mechanisms have emerged as indispensible tools for experimentally induced, sequence-specific inhibition of gene expression. The translation of these technologies for therapeutic applications has required the development of clinically feasible delivery strategies that facilitate the uptake of the siRNAs by specific cell type(s) and tissues. This feature review focuses on the progress made in the targeted delivery of siRNAs and the challenges encountered in the development of safe and effective therapeutic silencing agents.
Dykxhoorn
Derek M
DM
University of Miami Miller School of Medicine, John P Hussman Institute for Human Genomics, 1501 NW 10th Avenue, Miami, FL 33136, USA. DDykxhoorn@med.miami.edu
D000818:Animals; D005786:Gene Expression Regulation; D020868:Gene Silencing; D018390:Gene Targeting / Q000379:methods; D006801:Humans; D034622:RNA Interference; D034741:RNA, Small Interfering / Q000009:adverse effects / Q000627:therapeutic use*
D034741:RNA, Small Interfering