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...

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Published inJournal of pharmaceutical sciences Vol. 101; no. 11; pp. 4046 - 4066
Main Authors Guzman-Villanueva, Diana, El-Sherbiny, Ibrahim M., Herrera-Ruiz, Dea, Vlassov, Alexander V., Smyth, Hugh D.C.
Format Journal Article
LanguageEnglish
Published Hoboken Elsevier Inc 01.11.2012
Wiley Subscription Services, Inc., A Wiley Company
Wiley
American Pharmaceutical Association
Elsevier Limited
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
RNAi
RNA interference
formulations
in vivo knockdown
nonviral gene delivery
miRNA
siRNA
polymers
oligonucleotides
lipid
viral vectors
Pharmaceutical technology
Micro RNA
Lipids
Polymer
Genetic transfer
Gene silencing
In vivo
Formulation
Oligonucleotide
Gene therapy
Vector
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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
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ISSN:0022-3549
1520-6017
DOI:10.1002/jps.23300