Formulation Approaches to Short Interfering RNA and MicroRNA: Challenges and Implications

RNA interference has emerged as a potentially powerful tool in the treatment of genetic and acquired diseases by delivering short interfering RNA (siRNA) or microRNA (miRNA) to target genes, resulting in their silencing. However, many physicochemical and biological barriers have to be overcome to ob...

Full description

Saved in:
Bibliographic Details
Published in:Journal of pharmaceutical sciences 2012-11, Vol.101 (11), p.4046-4066
Main Authors: Guzman-Villanueva, Diana, El-Sherbiny, Ibrahim M., Herrera-Ruiz, Dea, Vlassov, Alexander V., Smyth, Hugh D.C.
Format: Article
Language:English
Subjects:
Biological and medical sciences
Chemistry, Pharmaceutical
formulations
formulations
polymers
Gene Silencing
General pharmacology
in vivo knockdown
lipid
Medical sciences
MicroRNAs - chemistry
miRNA
nonviral gene delivery
oligonucleotides
Pharmaceutical technology. Pharmaceutical industry
Pharmacology. Drug treatments
polymers
RNA Interference
RNA, Small Interfering - chemistry
RNAi
siRNA
viral vectors
Citations: Items that this one cites
Items that cite this one
Online Access:Get full text
Tags: Add Tag
No Tags, Be the first to tag this record!
Description
Summary:RNA interference has emerged as a potentially powerful tool in the treatment of genetic and acquired diseases by delivering short interfering RNA (siRNA) or microRNA (miRNA) to target genes, resulting in their silencing. However, many physicochemical and biological barriers have to be overcome to obtain efficient in vivo delivery of siRNA and miRNA molecules to the organ/tissue of interest, thereby enabling their effective clinical therapy. This review discusses the challenges associated with the use of siRNA and miRNA and describes the nonviral delivery strategies used in overcoming these barriers. More specifically, emphasis has been placed on those technologies that have progressed to clinical trials for both local and systemic siRNA and miRNA delivery. © 2012 Wiley Periodicals, Inc. and the American Pharmacists Association J Pharm Sci 101:4046–4066, 2012
ISSN:0022-3549
1520-6017
DOI:10.1002/jps.23300